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Clinical trials
catalyst calendar across sponsors →Studies where VRTX is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
233 interventional · 7 observational · 4 expanded access · 104 with posted results
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| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| A Long-term Follow-up Study in Participants Who Received CTX001NCT04208529New malignanciesOpen-label · Other | Phase 3 | Enrolling by invitation | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia | 160 | 2039-09-30 | in 4,790 d | 2026-08-11 |
| Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of AgeNCT05422222Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant MetabolitesNon-randomized · Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 210 | 2030-06-30 | in 1,411 d | 2026-05-28 |
| Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and OlderNCT05844449Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)Open-label · Treatment | Phase 3 | Enrolling by invitation | Drug: VNZ/TEZ/D-IVA | Cystic Fibrosis | 174 | 2029-07-30 | in 1,076 d | 2026-04-29 |
| A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia GravisNCT07501702Percent change in Total Immunoglobulin G (IgG)Randomized · Quadruple-masked · Treatment | Phase 2 | Recruiting | Drug: Povetacicept, Placebo | Myasthenia Gravis, Generalized | 30 | 2029-03-02 | in 926 d | 2026-08-03 |
| A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type INCT06926621Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 2 | Enrolling by invitation | Drug: VX-670 | Myotonic Dystrophy Type 1 (DM1) | 44 | 2029-01-30 | in 895 d | 2026-08-10 |
| Evaluation of Efficacy, Safety, and Tolerability of Povetacicept in Participants With Primary Membranous Nephropathy (pMN)NCT07204275Proportion of Participants with Complete Clinical Remission Definition 1 (CR1)Randomized · Quadruple-masked · Treatment | Phase 2/3 | Recruiting | Drug: Povetacicept, Tacrolimus | Primary Membranous Nephropathy | 176 | 2028-12-29 | in 863 d | 2026-08-03 |
| Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney DiseaseNCT05312879Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)Randomized · Quadruple-masked · Treatment | Phase 2/3 | Recruiting | Drug: VX-147, Placebo | Proteinuric Kidney Disease | 466 | 2028-06-02 | in 653 d | 2026-08-04 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)NCT05356195Proportion of Participants who Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)Open-label · Treatment | Phase 3 | Active, not recruiting | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies | 16 | 2027-11-14 | in 452 d | 2026-07-01 |
| Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and OlderNCT06460506Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 50 | 2027-09-30 | in 407 d | 2026-06-05 |
| Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)NCT07161037htTKV on MRI Over TimeOpen-label · Treatment | Phase 2 | Active, not recruiting | Drug: VX-407 | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 26 | 2027-07-22 | in 337 d | 2026-08-11 |
| A Safety, Tolerability, and Efficacy Study of VX-880 in Participants With Type 1 DiabetesNCT04786262Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)Open-label · Treatment | Phase 3 | Recruiting | Biological: VX-880 | Diabetes Mellitus, Type 1, Impaired Hypoglycemic Awareness, Severe Hypoglycemia | 52 | 2027-06-30 | in 315 d | 2026-06-30 |
| Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell DiseaseNCT05477563Fetal Hemoglobin (HbF) Concentration Over TimeOpen-label · Treatment | Phase 3 | Recruiting | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia | 26 | 2027-06-09 | in 294 d | 2026-03-23 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)NCT05329649Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12)Open-label · Treatment | Phase 3 | Active, not recruiting | Biological: CTX001 | Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases | 13 | 2027-06-06 | in 291 d | 2026-07-01 |
| Evaluation of Efficacy and Safety of Suzetrigine for Pain Associated With Diabetic Peripheral NeuropathyNCT06628908Change From Baseline in the Weekly Average of Daily Pain Intensity on the Numeric Pain Rating Scale (NPRS) at Week 12 Compared to PlaceboRandomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Drug: Suzetrigine, Placebo (matched to SUZ), Pregabalin, Placebo (matched to Pregabalin) | Diabetic Peripheral Neuropathic Pain | 1,100 | 2027-05-31 | in 285 d | 2026-06-15 |
| Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508delNCT05331183Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 297 | 2027-04-06 | in 230 d | 2026-08-11 |
| Evaluation of Efficacy and Safety of Suzetrigine (SUZ) for Pain Associated With Diabetic Peripheral NeuropathyNCT07231419Change From Baseline in the Weekly Average of Daily Pain Intensity on the Numeric Pain Rating Scale (NPRS) at Week 12 Compared to PlaceboRandomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Drug: Suzetrigine, Placebo (matched to SUZ) | Diabetic Peripheral Neuropathic Pain | 734 | 2027-04-06 | in 230 d | 2026-07-20 |
| Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral NeuropathyNCT06619860Change From Baseline in the Weekly Average of Daily Pain Intensity on the Numeric Pain Rating Scale (NPRS) at Week 12Randomized · Quadruple-masked · Treatment | Phase 2 | Recruiting | Drug: VX-993, Pregabalin, Placebo (matched to pregabalin), Placebo (matched to VX-993) | Diabetic Peripheral Neuropathic Pain | 300 | 2027-03-25 | in 218 d | 2026-06-01 |
| Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-272 in Healthy ParticipantsNCT07437105Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Recruiting | Drug: VX-272, Placebo | Cystic Fibrosis | 128 | 2027-03-23 | in 216 d | 2026-03-31 |
| A Safety, Tolerability, and Efficacy Study of VX-264 in Participants With Type 1 DiabetesNCT05791201Part A and Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 1/2 | Active, not recruiting | Drug: VX-264 | Type 1 Diabetes | 7 | 2027-03-12 | in 205 d | 2026-06-04 |
| A Phase 1, First-in-human Study of VX-433NCT07584434Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Recruiting | Drug: VX-433, Placebo, Midazolam, Bupropion | Narcolepsy Type 1 (NT1) | 118 | 2027-03-05 | in 198 d | 2026-06-08 |
| Evaluation of the Excretion of Suzetrigine Into Breast Milk in Healthy Lactating Female ParticipantsNCT07378865Total Amount of SUZ in Breast Milk of Lactating Female ParticipantsOpen-label · Treatment | Phase 1 | Recruiting | Drug: Suzetrigine | Pain | 12 | 2027-02-26 | in 191 d | 2026-05-13 |
| A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)NCT06185764Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)Randomized · Quadruple-masked · Treatment | Phase 1/2 | Recruiting | Drug: VX-670, Placebo | Myotonic Dystrophy Type 1 (DM1) | 52 | 2027-02-02 | in 167 d | 2026-06-08 |
| Evaluation of Pain Treatment After Total Knee ArthroplastyNCT07538570Total Opioid UsageNon-randomized · Quadruple-masked · Treatment | Phase 4 | Recruiting | Drug: Suzetrigine, Placebo | Pain | 60 | 2027-01-30 | in 164 d | 2026-08-18 |
| Evaluation of the Long-term Safety and Effectiveness of Suzetrigine (SUZ) in Participants With Painful Diabetic Peripheral Neuropathy (DPN)NCT06696443Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: Suzetrigine | Diabetic Peripheral Neuropathic Pain | 455 | 2027-01-25 | in 159 d | 2026-05-18 |
| Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney DiseaseNCT06747572observationalPart A: Percentage of PKD1 Gene Variant Groups in the ADPKD Population | — | Active, not recruiting | — | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 401 | 2026-12-31 | in 134 d | 2026-07-21 |
| Inaxaplin in Participants With Proteinuric APOL1 Mediated Kidney Disease With or Without ComorbiditiesNCT06794996Percent Change in Urine Albumin-Creatinine Ratio (UACR)Open-label · Treatment | Phase 2 | Active, not recruiting | Drug: Inaxaplin | Proteinuric Kidney Disease | 42 | 2026-12-30 | in 133 d | 2026-05-05 |
| Dose Escalation Study Evaluating the Safety and Pharmacokinetics of VX-581 in Healthy ParticipantsNCT07283770Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Recruiting | Drug: VX-581, Placebo, D-IVA, TEZ | Cystic Fibrosis | 136 | 2026-11-19 | in 92 d | 2026-07-16 |
| A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination TherapyNCT05444257Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 3 | Active, not recruiting | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 822 | 2026-10-30 | in 72 d | 2026-07-21 |
| An Efficacy, Safety, and Tolerability Study of VX-880 in Participants With Type 1 Diabetes With a Kidney TransplantNCT06832410Proportion of Participants who are Insulin IndependentOpen-label · Treatment | Phase 3 | Recruiting | Biological: Biological/Vaccine | Type 1 Diabetes, Kidney Transplant | 10 | 2026-09-17 | in 29 d | 2026-03-23 |
| A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF)NCT05668741Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 1/2 | Active, not recruiting | Drug: VX-522 mRNA therapy, IVA | Cystic Fibrosis | 26 | 2026-04-21actual | — | 2026-05-22 |
| Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC GenotypeNCT05951205Proportion of Participants with an Average Fetal Hemoglobin (HbF) Greater Than or Equal To (>=) 20 percent (%) on or After 6 MonthsOpen-label · Treatment | Phase 3 | Withdrawnsponsor's stated reason: Modification to the clinical development plan | Biological: Exa-cel | Sickle Cell Disease | 0 | 2033-12-31 | in 2,691 d | 2026-03-25 |
| Effects of Efavirenz on the Pharmacokinetics of Suzetrigine in Healthy ParticipantsNCT07570069Maximum Observed Plasma Concentration (Cmax) of SUZ and its Metabolite in the Absence and Presence of EfavirenzOpen-label · Treatment | Phase 1 | Completed | Drug: Suzetrigine, Efavirenz | Pain | 18 | 2026-07-16actual | — | 2026-08-18 |
| Evaluation of VX-828 in Healthy Participants and in Participants With Cystic FibrosisNCT06154447Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-828, Placebo, Itraconazole, Midazolam, Tezacaftor, VX-118, Placebo, Deutivacaftor, VX-828 | Cystic Fibrosis | 165 | 2026-07-10actual | — | 2026-08-10 |
| Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy ParticipantsNCT07349394Maximum Observed Concentration (Cmax) of Rosuvastatin in the Absence and Presence of VNZ/TEZ/D-IVAOpen-label · Treatment | Phase 1 | Completed | Drug: VNZ/TEZ/D-IVA, Rosuvastatin | Cystic Fibrosis | 18 | 2026-02-28actual | — | 2026-04-02 |
| A Phase 1 Study Evaluating Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy ParticipantsNCT07022119Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-407, Placebo | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 32 | 2026-02-24actual | — | 2026-03-27 |
| Effects of VX-407 on the Pharmacokinetics of Oral Contraceptives in Healthy ParticipantsNCT07074327Part A: Maximum Observed Plasma Concentration (Cmax) of LNG and EE in the Absence and Presence of VX-407Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-407, LNG/EE, NGM/EE, NET/EE, DRSP/EE | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 74 | 2026-02-20actual | — | 2026-03-30 |
| Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and OlderNCT05153317Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 71 | 2026-01-29actual | — | 2026-03-03 |
| Natural History Study of Exocrine Pancreatic Function in Infants With Cystic Fibrosis (CF)NCT06506773observationalProportion of Participants with FE-1 greater than or equal to (≥) 200 microgram per gram (µg/g) Over Time | — | Completed | — | Cystic Fibrosis | 79 | 2025-11-27actual | — | 2025-12-31 |
| A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-ThalassemiaNCT03655678Proportion of participants achieving transfusion independence for at least 12 consecutive months (TI12)Open-label · Treatment | Phase 2/3 | Completed | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies | 59 | 2025-11-13actual | — | 2025-12-17 |
| A Single-arm Study Evaluating the Effectiveness and Safety of Suzetrigine (SUZ) for Acute Pain After Selected SurgeriesNCT06887959Proportion of Participants Reporting Good, Very Good, or Excellent on a Patient Global Assessment (PGA) for Pain Control at the End of TreatmentOpen-label · Treatment | Phase 4 | Completed | Drug: Suzetrigine | Acute Pain | 47 | 2025-10-27actual | — | 2025-12-15 |
| A Single-Arm Study Evaluating the Effectiveness and Safety of Suzetrigine (SUZ) for Acute Pain After Aesthetic or Reconstructive SurgeriesNCT06887972Proportion of Participants Reporting Good, Very Good, or Excellent on a Patient Global assessment (PGA) for Pain Control at the end of TreatmentOpen-label · Treatment | Phase 4 | Completed | Drug: Suzetrigine | Acute Pain | 100 | 2025-10-24actual | — | 2025-12-15 |
| Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of AgeNCT05882357Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant MetabolitesNon-randomized · Open-label · Treatment | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 70 | 2025-09-04actual | — | 2025-10-06 |
| A Study to Evaluate the Relative Bioavailability of Povetacicept Formulations and Bioequivalence of Povetacicept PresentationsNCT07010406Area Under the Concentration Versus Time Curve From the Time of Dosing to 28 Days (AUC0-28d) of Free PovetaciceptRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: Povetacicept | Healthy Volunteers | 136 | 2025-08-19actual | — | 2025-09-18 |
| A Phase 1 Dose Escalation Study of VX-973 in Healthy ParticipantsNCT06615570Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-973, Placebo | Pain | 30 | 2025-07-21actual | — | 2025-08-26 |
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell DiseaseNCT03745287Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12)Open-label · Treatment | Phase 2/3 | Completed | Biological: CTX001 | Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies | 63 | 2025-07-07actual | — | 2025-08-11 |
| A Study Evaluating Efficacy and Safety of VX-993 for Acute Pain After a BunionectomyNCT06619847Time-weighted Sum of Pain Intensity Difference (SPID) as Recorded on the Numeric Pain Rating Scale (NPRS) From 0 to 48 Hours (SPID48)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: VX-993, HB/APAP, Placebo (matched to VX-993), Placebo (matched to HB/APAP) | Acute Pain | 367 | 2025-05-27actual | — | 2026-05-28 |
| A Study to Evaluate How the Body Processes 14C-VX-993 After a Single Oral DoseNCT06886269Terminal Phase Rate Constant (λz) of VX-993 in PlasmaOpen-label · Treatment | Phase 1 | Completed | Drug: 14C-VX-993 | Pain | 8 | 2025-05-17actual | — | 2025-06-18 |
| A Phase 1 Study to Evaluate the Relative Bioavailability of a VX-828 Tablet FormulationNCT06861413Area Under the Concentration Versus Time Curve from the Time of dosing to the Last Measurable Concentration (AUC0-tlast) of VX-828 in PlasmaRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-828, VX-828 | Cystic Fibrosis | 68 | 2025-05-05actual | — | 2025-06-04 |
| Evaluation of the Effect of Suzetrigine (SUZ) on the Pharmacokinetics of Oral Contraceptives in Healthy Female ParticipantsNCT06820307Part A: Maximum Observed Plasma Concentration (Cmax) of DRSP and EE in the Absence and Presence of SUZNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Suzetrigine, DRSP/EE, NGM/EE | Pain | 50 | 2025-04-29actual | — | 2025-05-29 |
| A Study of Suzetrigine (SUZ) Spray-dried Dispersion (SSD) in Healthy Adult PanelistsNCT06834009Flavor Assessment Based on Flavor Profile Method of Descriptive Sensory Analysis per American Society for Testing and Material (ASTM) Approved Sensory Evaluation MethodOpen-label · Treatment | Phase 1 | Completed | Drug: SUZ | Pain | 10 | 2025-04-24actual | — | 2025-05-14 |
| A Microneurography Study of NaV1.8 Inhibition in Healthy AdultsNCT06420765Change From Baseline in Activity Dependent Slowing (ADS) Over TimeRandomized · Quadruple-masked · Basic science | Phase 1 | Completed | Drug: VX-150, VX-548, VX-993, Placebo | Pain | 92 | 2025-03-22actual | — | 2025-04-20 |
| A Phase 1 Dose Escalation Study of Intravenous VX-993 in Healthy AdultsNCT06394167Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-993, Placebo | Pain | 88 | 2025-02-18actual | — | 2025-03-20 |
| A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of VX-407 in Healthy ParticipantsNCT06345755Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-407, Placebo, Midazolam, VX-407 | Autosomal Dominant Polycystic Kidney Disease (ADPKD) | 159 | 2025-01-27actual | — | 2025-07-14 |
| Evaluation of the Pharmacokinetic Drug-drug Interactions Between VX-993 and Metformin in Healthy AdultsNCT06523595Maximum Observed Plasma Concentration (Cmax) of Metformin in Absence and Presence of VX-993Open-label · Treatment | Phase 1 | Completed | Drug: VX-993, Metformin | Pain | 31 | 2024-12-31actual | — | 2025-01-31 |
| Evaluation of the Pharmacokinetics and Safety of Inaxaplin in Participants With Mild or Moderate Hepatic ImpairmentNCT06529796Maximum Observed Plasma Concentration (Cmax) of IXPNon-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: IXP | Focal Segmental Glomerulosclerosis (FSGS) | 36 | 2024-12-20actual | — | 2025-01-20 |
| Evaluation of Efficacy and Safety of VX-548 for Painful Lumbosacral Radiculopathy (PLSR)NCT06176196Change From Baseline in the Weekly Average of Daily leg Pain Intensity on a Numeric Pain Rating Scale (NPRS)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: VX-548, Placebo | Painful Lumbosacral Radiculopathy | 218 | 2024-10-02actual | — | 2025-10-01 |
| A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation of VX-993NCT06508762Maximum Observed Plasma Concentration (Cmax) of VX-993Open-label · Treatment | Phase 1 | Completed | Drug: VX-993 | Pain | 24 | 2024-09-10actual | — | 2024-10-10 |
| A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ GenotypeNCT05643495results2025-09-08Change in Blood Functional Alpha-1 Antitrypsin (AAT) LevelsNon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Study drug dosing was permanently discontinued at the Sponsor's discretion based on frequency and nature of skin and subcutaneous tissue disorders. | Drug: VX-864 | Alpha-1 Antitrypsin Deficiency | 14 | 2024-08-19actual | — | 2025-09-08 |
| A Phase 1, Open-label Study Evaluating the Pharmacokinetics and Drug-drug Interaction of VX-993 in Healthy AdultsNCT06392659Part A: Maximum Observed Plasma Concentration (Cmax) of Midazolam in Absence or Presence of VX-993Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-993, Gemfibrozil, Itraconazole, Midazolam | Pain | 42 | 2024-07-02actual | — | 2024-08-01 |
| A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation and Strength of SuzetrigineNCT06336096Part A: Maximum Observed Plasma Concentration (Cmax) of SUZRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: Suzetrigine, Suzetrigine | Pain | 48 | 2024-06-19actual | — | 2024-07-19 |
| A Study to Evaluate Pharmacokinetics (PK) and Safety of Inaxaplin in Participants With Renal ImpairmentNCT05865171Maximum Observed Plasma Concentration (Cmax) of IXPRandomized · Open-label · Basic science | Phase 1 | Completed | Drug: IXP | Renal Impairment | 16 | 2024-06-12actual | — | 2024-07-12 |
| A Phase 1 Study Evaluating the Safety, Pharmacokinetics, and Pharmacodynamics, of VX-993NCT06226454Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-993, Placebo | Pain | 39 | 2024-06-12actual | — | 2024-07-12 |
| A Study To Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor/Tezacaftor/Deutivacaftor(VNZ/TEZ/D-IVA)NCT06299709Part A: Maximum Observed Plasma Concentration (Cmax) of VNZ, TEZ, and D-IVARandomized · Open-label · Treatment | Phase 1 | Completed | Drug: VNZ/TEZ/D-IVA, VNZ/TEZ/D-IVA | Cystic Fibrosis | 34 | 2024-05-23actual | — | 2025-09-26 |
| A Study to Evaluate the Relative Bioavailability and Food Effect of a VX-118 Tablet FormulationNCT06312787Maximum Observed Plasma Concentration (Cmax) of VX-118Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-118 | Cystic Fibrosis | 15 | 2024-05-04actual | — | 2024-06-03 |
| A Study of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) in Healthy Adult PanelistsNCT06299696Flavor Assessment Based on Flavor Profile Method of Descriptive Sensory Analysis per American Society for Testing and Material (ASTM) Approved Sensory Evaluation MethodOpen-label · Basic science | Phase 1 | Completed | Drug: VNZ/TEZ/D-IVA | Cystic Fibrosis | 10 | 2024-04-16actual | — | 2024-05-16 |
| A Phase 1 Dose Escalation Study of VX-973 in Healthy AdultsNCT05866055Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-973, Placebo, Midazolam | Pain | 30 | 2024-04-04actual | — | 2024-05-06 |
| Evaluation of the Pharmacokinetics and Safety of VX-548 in Participants With Renal ImpairmentNCT05704556Maximum Observed Plasma Concentration (Cmax) of VX-548 and its MetaboliteNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-548 | Pain | 38 | 2024-03-30actual | — | 2024-04-29 |
| Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and OlderNCT04183790results2025-05-18Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 64 | 2024-02-24actual | — | 2025-05-18 |
| Evaluation of Efficacy and Safety of VX-548 for Acute Pain After a BunionectomyNCT05553366results2025-08-01Time-weighted Sum of the Pain Intensity Difference (SPID) as Recorded on a Numeric Pain Rating Scale (NPRS) From 0 to 48 Hours (SPID48), SUZ Compared to PlaceboRandomized · Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: SUZ, HB/APAP, Placebo (matched to SUZ), Placebo (matched to HB/APAP) | Acute Pain | 1,075 | 2023-12-15actual | — | 2025-08-01 |
| A Single-arm Study to Evaluate Safety and Effectiveness of VX-548 for Acute PainNCT05661734results2025-07-01Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Suzetrigine | Pain | 258 | 2023-12-12actual | — | 2025-07-01 |
| A Study to Evaluate the Effect of Food on the Pharmacokinetics of VX-548NCT05851157Maximum Observed Plasma Concentration (Cmax) of VX-548 and its MetaboliteOpen-label · Treatment | Phase 1 | Completed | Drug: VX-548 | Pain | 73 | 2023-10-30actual | — | 2024-03-20 |
| Evaluation of Efficacy and Safety of VX-548 for Painful Diabetic Peripheral Neuropathy (DPN)NCT05660538results2025-07-01Change From Baseline in the Weekly Average of Daily Pain Intensity on a Numeric Pain Rating Scale (NPRS)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Suzetrigine, Pregabalin, Placebo (matched to SUZ), Placebo (matched to pregabalin) | Diabetic Peripheral Neuropathy | 194 | 2023-10-12actual | — | 2025-07-01 |
| A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive MutationNCT03277196results2024-10-23Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEsNon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: IVA | Cystic Fibrosis | 86 | 2023-10-02actual | — | 2024-10-23 |
| A Phase 1 Dose Escalation Study of VX-993 in Healthy ParticipantsNCT05653323Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-993, Placebo | Pain | 70 | 2023-09-12actual | — | 2023-10-12 |
| A Phase 1, First-in-human Study of VX-668NCT05727800Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-668, Placebo | Alpha-1 Antitrypsin Deficiency | 114 | 2023-09-12actual | — | 2024-03-15 |
| A Study Evaluating the Relative Bioavailability and Food Effect of a Tablet Formulation of VX-147NCT05955872Maximum Observed Plasma Concentration (Cmax) of VX-147 Test Compared to VX-147 ReferenceRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-147 | Focal Segmental Glomerulosclerosis (FSGS) | 21 | 2023-09-02actual | — | 2023-09-13 |
| Evaluation of Efficacy and Safety of Suzetrigine for Acute Pain After an AbdominoplastyNCT05558410results2025-07-01Time-weighted Sum of the Pain Intensity Difference (SPID) as Recorded on a Numeric Pain Rating Scale (NPRS) From 0 to 48 Hours (SPID48) SUZ Compared to PlaceboRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Suzetrigine (SUZ), HB/APAP, Placebo (matched to SUZ), Placebo (matched to HB/APAP) | Acute Pain | 1,118 | 2023-08-25actual | — | 2025-07-01 |
| Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment InitiationNCT04235140results2024-09-19Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: LUM/IVA | Cystic Fibrosis | 52 | 2023-08-22actual | — | 2024-09-19 |
| Evaluation of the Pharmacokinetics and Safety of VX-548 in Participants With Mild or Moderate Hepatic ImpairmentNCT05560464Maximum Observed Plasma Concentration (Cmax) of VX-548Non-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: VX-548 | Pain | 36 | 2023-07-24actual | — | 2024-03-20 |
| Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del MutationNCT05274269results2024-08-01Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA · Other: Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) | Cystic Fibrosis | 307 | 2023-07-05actual | — | 2024-08-01 |
| An Electrocardiogram Study to Evaluate the Effect of Vanzacaftor on the QT/QTc Interval in Healthy ParticipantsNCT05867147Change in QT interval corrected by Fridericia's formula (QTcF)Randomized · Double-masked · Treatment | Phase 1 | Completed | Drug: Vanzacaftor, Vanzacaftor Placebo, Moxifloxacin, Moxifloxacin Placebo | Cystic Fibrosis | 56 | 2023-06-14actual | — | 2023-07-14 |
| A Study to Evaluate the Effect of VX-548 on the QT/QTc Interval in Healthy ParticipantsNCT05818852Change in QT interval corrected by Fridericia's formula (QTcF)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-548, Moxifloxacin, Moxifloxacin Placebo, VX-548 Placebo | Pain | 72 | 2023-06-12actual | — | 2024-03-20 |
| Evaluation of the Effects of Omeprazole and Rifampin on the Pharmacokinetics of VX-548 in Healthy ParticipantsNCT05635110Part A: Maximum Observed Plasma Concentration (Cmax) of VX-548 and its Metabolite in the Absence and Presence of OmeprazoleNon-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: VX-548, Omeprazole, Rifampin | Pain | 31 | 2023-06-08actual | — | 2024-03-20 |
| A Phase 1, First-in-human Study of VX-634NCT05579431Part A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-634, Placebo | Alpha 1-Antitrypsin Deficiency | 127 | 2023-05-24actual | — | 2024-03-12 |
| A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del MutationNCT05076149results2024-06-13Absolute Change in Percent Predicted Forced Expiratory Volume in 1second (ppFEV1)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) | Cystic Fibrosis | 597 | 2023-05-18actual | — | 2024-06-13 |
| A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF)NCT05033080results2024-06-10Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, Placebo (matched to VX-121/TEZ/D-IVA), Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) | Cystic Fibrosis | 435 | 2023-05-12actual | — | 2024-10-03 |
| A Study to Evaluate the Pharmacokinetic Drug-drug Interactions Between VX-548, Midazolam, and DigoxinNCT05541471Maximum Observed Plasma Concentration (Cmax) of Midazolam in the Absence and Presence of VX-548Open-label · Basic science | Phase 1 | Completed | Drug: VX-548, Midazolam, Digoxin | Pain | 37 | 2023-04-16actual | — | 2024-03-20 |
| A Phase 1 Dose Escalation Study of Oral VX-708 in Healthy ParticipantsNCT05347394Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-708, Placebo | Acute Pain | 100 | 2023-04-14actual | — | 2023-05-15 |
| A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF GenotypesNCT04545515results2024-05-08Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 120 | 2023-03-24actual | — | 2024-05-08 |
| Pharmacokinetics and Safety Assessment of VX-121/Tezacaftor/Deutivacaftor in Participants With Moderate Hepatic ImpairmentNCT05437120Maximum Observed Plasma Concentration (Cmax) of VX-121, TEZ, D-IVA, and Relevant MetabolitesNon-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 16 | 2023-03-16actual | — | 2023-03-30 |
| A Phase 1 Dose Escalation Study of VX-708 in Healthy ParticipantsNCT05518734Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-708, Itraconazole, Midazolam, Placebo | Pain | 64 | 2023-03-07actual | — | 2023-03-30 |
| A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyNCT03525574results2024-05-08Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 507 | 2023-01-09actual | — | 2025-09-24 |
| A Study Evaluating the Long-Term Safety of Elexacaftor Combination TherapyNCT04362761results2023-07-28Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 172 | 2022-12-21actual | — | 2023-07-28 |
| A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF)NCT05111145results2023-07-11Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 86 | 2022-12-20actual | — | 2023-07-11 |
| Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyNCT04058366results2024-01-16Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 251 | 2022-12-16actual | — | 2024-01-16 |
| A Study Evaluating the Long-term Safety of VX-445 Combination TherapyNCT04043806results2023-07-06Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 458 | 2022-12-14actual | — | 2023-07-06 |
| A Study to Evaluate the Relative Bioavailability of a Fixed-dose Combination Tablet of VX-121/Tezacaftor/DeutivacaftorNCT05535959Maximum Observed Plasma Concentration (Cmax) of VX-121, TEZ, and D-IVARandomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 16 | 2022-11-09actual | — | 2022-12-12 |
| A Phase 1 Dose Escalation Study to Evaluate Safety and Pharmacokinetics (PK) of VX-840 in Healthy ParticipantsNCT05324410Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-840, Placebo | APOL1-mediated Kidney Disease | 88 | 2022-11-07actual | — | 2022-12-08 |
| A Study to Evaluate the Relative Bioavailability and Food Effect of a New Tablet Formulation of VX-548NCT05455502Maximum Observed Plasma Concentration (Cmax) of VX-548Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-548 | Pain | 24 | 2022-09-17actual | — | 2024-03-20 |
| A Microneurography (MNG) Study of VX-150 in Healthy ParticipantsNCT05418712Change From Baseline in Action Potential (AP) Latency at 0.25 Hertz (Hz) Over TimeRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: Placebo, VX-150 | Pain | 45 | 2022-09-14actual | — | 2022-10-14 |
| A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)NCT04969224results2023-10-03Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 82 | 2022-07-26actual | — | 2025-09-24 |
| A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)NCT04599465results2023-08-03Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 69 | 2022-07-14actual | — | 2023-08-03 |
| A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR MutationNCT02725567results2023-09-07Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)Non-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: IVA | Cystic Fibrosis | 57 | 2022-06-28actual | — | 2023-09-07 |
| Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 YearsNCT04537793results2023-06-28Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant MetabolitesNon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 83 | 2022-06-03actual | — | 2023-06-28 |
| A Study Evaluating Efficacy and Safety of VX-548 for Acute Pain After a BunionectomyNCT04977336results2025-06-25Time-Weighted Sum of Pain Intensity Difference (SPID) as Recorded on a Numeric Pain Rating Scale (NPRS) 0 to 48 Hours (SPID48) After the First Dose of Study DrugRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-548, HB/APAP, Placebo (matched to VX-548), Placebo (matched to HB/APAP) | Acute Pain | 274 | 2022-02-17actual | — | 2025-06-25 |
| A Decentralized Study to Evaluate Physical Activity and Cough Frequency Using Wearable Technology in Cystic FibrosisNCT04923464observationalCompliance With Actigraphy Wearable Device Measured as Percentage of Time a Participant is Wearing the Actigraphy Wearable Device | — | Completed | — | Cystic Fibrosis | 51 | 2021-12-20actual | — | 2022-01-19 |
| A Study Evaluating Efficacy and Safety of VX-548 for Acute Pain After an AbdominoplastyNCT05034952results2024-12-27Time-weighted Sum of the Pain Intensity Difference (SPID) as Recorded on a Numeric Pain Rating Scale (NPRS) at Rest 0 to 48 Hours (SPIDr0-48) After the First Dose of Study DrugRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-548, HB/APAP, Placebo (matched to VX-548), Placebo (matched to HB/APAP) | Acute Pain | 303 | 2021-12-05actual | — | 2024-12-27 |
| Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental GlomerulosclerosisNCT04340362results2023-07-10Percent Change From Baseline in UPCROpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-147 | Glomerulosclerosis, Focal Segmental | 16 | 2021-11-11actual | — | 2025-09-24 |
| Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508delNCT03601637results2023-01-06Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVANon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: LUM, IVA | Cystic Fibrosis | 61 | 2021-10-29actual | — | 2023-01-06 |
| A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF GenotypesNCT04353817results2022-07-26Absolute Change in Lung Clearance Index 2.5 (LCI2.5)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA · Other: Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) | Cystic Fibrosis | 121 | 2021-05-17actual | — | 2022-07-26 |
| Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ GenotypeNCT04474197results2022-05-26Change in Plasma Functional Alpha-1 Antitrypsin (AAT) LevelsRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-864, Placebo | Alpha1-Antitrypsin Deficiency | 44 | 2021-05-04actual | — | 2022-05-26 |
| Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ GenotypeNCT04167345results2022-02-02Change in Plasma Functional Alpha-1 Antitrypsin (AAT) LevelsRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: at Sponsor's discretion | Drug: VX-814, Placebo | Alpha 1-Antitrypsin Deficiency | 48 | 2020-11-14actual | — | 2022-02-02 |
| Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)NCT02445053observationalPulmonary exacerbations - Number of pulmonary exacerbations and duration of treatment for pulmonary exacerbations during Kalydeco treatment compared to the period before Kalydeco treatment | — | Completed | Drug: ivacaftor | Cystic Fibrosis | 75 | Oct 2020actual | — | 2020-11-13 |
| A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR MutationNCT03537651results2021-11-26Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: TEZ/IVA, IVA | Cystic Fibrosis | 130 | 2020-10-28actual | — | 2024-04-19 |
| A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508delNCT03625466results2021-12-08Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: LUM/IVA, LUM/IVA, Placebo | Cystic Fibrosis | 51 | 2020-10-09actual | — | 2022-11-02 |
| A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination TherapyNCT03447262results2022-01-25Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Terminatedsponsor's stated reason: At Sponsor's Discretion | Drug: VX-659/TEZ/IVA, IVA | Cystic Fibrosis | 484 | 2020-09-09actual | — | 2022-01-25 |
| A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic FibrosisNCT03911713results2022-01-25Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-561, IVA, Placebo, Placebo | Cystic Fibrosis | 77 | 2020-08-20actual | — | 2022-01-25 |
| Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of AgeNCT03691779results2021-10-22Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVANon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 71 | 2020-08-07actual | — | 2021-10-22 |
| A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508delNCT04105972results2021-08-18Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain ScoreRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, TEZ/IVA, IVA | Cystic Fibrosis | 176 | 2020-07-24actual | — | 2021-08-18 |
| A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)NCT04058353results2021-07-02Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for ELX/TEZ/IVA GroupRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA, TEZ/IVA | Cystic Fibrosis | 271 | 2020-06-12actual | — | 2021-07-02 |
| A Study to Confirm the Long-term Safety and Effectiveness of Kalydeco in Patients With Cystic Fibrosis Who Have an R117H-CFTR Mutation, Including Pediatric PatientsNCT02722057observationalLung function measurements (percent predicted forced expiratory volume in 1 second [FEV1] and forced vital capacity [FVC]) | — | Completed | — | Cystic Fibrosis | 368 | Dec 2019actual | — | 2020-02-05 |
| A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic FibrosisNCT03912233results2023-04-20Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-121, TEZ, VX-561, TEZ/IVA, IVA, Placebo | Cystic Fibrosis | 87 | 2019-12-10actual | — | 2023-04-20 |
| A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR MutationNCT03125395results2020-08-07Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: LUM/IVA | Cystic Fibrosis | 57 | 2019-07-17actual | — | 2020-08-07 |
| A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR MutationNCT02565914results2020-06-16Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: TEZ/IVA, IVA | Cystic Fibrosis | 1,131 | May 2019actual | — | 2023-09-28 |
| Study of VX-121 in Healthy Subjects and in Subjects With Cystic FibrosisNCT03768089results2022-07-14Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: Placebo (matched to VX-121 suspension), VX-121 (Suspension), TEZ/IVA, IVA, Placebo (matched to TEZ/IVA), Placebo (matched to IVA), VX-121 (Tablet), Placebo (matched to VX-121 tablet) | Cystic Fibrosis | 115 | 2019-05-03actual | — | 2022-07-14 |
| A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)NCT03525444results2020-05-19Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-445/TEZ/IVA, IVA, Placebo | Cystic Fibrosis | 405 | 2019-04-24actual | — | 2020-05-19 |
| A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)NCT03447249results2020-03-13Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-659/TEZ/IVA, IVA, Placebo | Cystic Fibrosis | 385 | 2019-02-05actual | — | 2020-03-13 |
| Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of AgeNCT03633526results2020-02-05Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVAOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Terminatedsponsor's stated reason: Study discontinued after Part A due to Sponsor's discretion. | Drug: VX-659/TEZ/IVA, IVA | Cystic Fibrosis | 18 | 2019-01-18actual | — | 2020-02-05 |
| A Dose-ranging Study to Evaluate Efficacy and Safety of VX-150 in Subjects With Acute Pain Following BunionectomyNCT03764072results2022-02-09Time-weighted Sum of the Pain Intensity Difference as Recorded on Numeric Pain Rating Scale (NPRS) 0 to 24 Hours (SPID24) After First DoseRandomized · Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-150, Placebo | Acute Pain | 250 | 2019-01-17actual | — | 2022-02-09 |
| A Study of VX-445 Combination Therapy in CF Subjects Homozygous for F508del (F/F)NCT03525548results2020-01-27Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-445/TEZ/IVA, TEZ/IVA, IVA, Placebo, Placebo | Cystic Fibrosis | 113 | 2018-12-28actual | — | 2020-01-27 |
| A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic FibrosisNCT03559062results2020-02-11Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: TEZ/IVA, IVA, Placebo, Placebo | Cystic Fibrosis | 67 | 2018-12-21actual | — | 2020-02-11 |
| A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR MutationNCT03068312results2020-01-06Change in Lung Clearance Index 2.5 (LCI2.5)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Ivacaftor, Placebo | Cystic Fibrosis | 38 | 2018-12-18actual | — | 2020-02-26 |
| Study to Assess the Efficacy and Safety of VX-210 in Subjects With Acute Traumatic Cervical Spinal Cord InjuryNCT02669849results2020-01-22Change in Upper Extremity Motor Score (UEMS)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2/3 | Terminated | Drug: VX-210, Placebo | Cervical Spinal Cord Injury | 70 | Nov 2018actual | — | 2020-01-22 |
| A Study to Evaluate the Efficacy and Safety of VX-150 in Treating Subjects With Pain Caused by Small Fiber NeuropathyNCT03304522results2021-11-15Change in Weekly Average of Daily Pain Intensity on the 11 Point NRSRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-150, Placebo | Small Fiber Neuropathy | 89 | 2018-10-12actual | — | 2021-11-15 |
| A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)NCT02953314results2019-12-05Part A: Maximum Observed Concentration (Cmax) of TEZ and IVANon-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: TEZ, TEZ/IVA, IVA | Cystic Fibrosis | 83 | Sep 2018actual | — | 2020-03-04 |
| A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)NCT03460990results2019-10-17Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-659/TEZ/IVA, TEZ/IVA, IVA, Placebo, Placebo | Cystic Fibrosis | 116 | 2018-09-26actual | — | 2019-10-17 |
| Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With IvacaftorNCT02544451results2019-10-29Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Non-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: LUM/IVA, LUM/IVA | Cystic Fibrosis | 246 | Aug 2018actual | — | 2021-05-24 |
| A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)NCT03150719results2019-09-12Incidence of Respiratory Adverse Events of Special Interest (RAESIs)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Tezacaftor/Ivacaftor, Ivacaftor, Placebo, Placebo | Cystic Fibrosis | 98 | 2018-08-09actual | — | 2019-09-12 |
| A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR MutationNCT02730208results2019-08-21Absolute Change in Total Brody/CF-CT ScoreRandomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Tezacaftor/Ivacaftor, Ivacaftor, Placebo, Placebo | Cystic Fibrosis | 41 | Jul 2018actual | — | 2019-10-23 |
| A Study of VX-445 in Healthy Subjects and Subjects With Cystic FibrosisNCT03227471results2022-01-18Parts A, B and C: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 1/2 | Completed | Drug: IVA, TEZ/IVA, VX-445, Matched Placebo, TEZ, VX-561, VX-445 | Cystic Fibrosis | 225 | 2018-03-27actual | — | 2022-01-18 |
| A Study Evaluating the Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic FibrosisNCT03224351results2021-04-22Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-659, TEZ/IVA, IVA, Placebo (matched to VX-659/TEZ/IVA), TEZ, VX-561, Placebo (matched to VX-659/TEZ/VX-561) | Cystic Fibrosis | 124 | 2018-02-28actual | — | 2021-04-22 |
| A Study Evaluating the Safety of VX-152 Combination Therapy in Adults With Cystic FibrosisNCT02951195results2021-01-28Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-152, TEZ/IVA, IVA, Placebo, Placebo | Cystic Fibrosis | 80 | Jan 2018actual | — | 2021-01-28 |
| A Study to Evaluate Efficacy and Safety of VX-150 in Subjects With Acute Pain Following BunionectomyNCT03206749results2021-02-03Time-weighted Sum of the Pain Intensity Difference (SPID) Between VX-150 Versus Placebo as Recorded on a Numeric Pain Rating Scale (NPRS) 0 to 24 Hours After the First DoseRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: VX-150, HB/APAP, Placebo | Acute Pain | 243 | 2017-12-01actual | — | 2021-02-03 |
| A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to IvacaftorNCT02412111results2019-01-08Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Ivacaftor, Tezacaftor/Ivacaftor | Cystic Fibrosis | 156 | Sep 2017actual | — | 2019-01-08 |
| Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508delNCT02797132results2018-10-30Part A: Pre-dose Concentration (Ctrough) of LUM and IVAOpen-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: LUM/IVA | Cystic Fibrosis | 62 | Sep 2017actual | — | 2018-10-30 |
| A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR MutationNCT02875366results2019-06-17Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24Randomized · Triple-masked · OtherStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 4 | Completed | Drug: LUM/IVA, Placebo | Cystic Fibrosis | 70 | Sep 2017actual | — | 2019-06-17 |
| Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR MutationNCT03061331results2018-10-02Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 8Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: LUM/IVA, Placebo | Cystic Fibrosis | 20 | 2017-09-06actual | — | 2018-10-02 |
| A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating MutationNCT02742519results2018-11-19Absolute Change From Baseline in Lung Clearance Index (LCI2.5) Through 8 Weeks of Treatment (Average of Week 4 and Week 8 LCI2.5)Randomized · Double-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Terminatedsponsor's stated reason: Low enrollment. | Drug: ivacaftor, Placebo | Cystic Fibrosis | 14 | Aug 2017actual | — | 2018-11-19 |
| A Pilot Study to Evaluate the Use of Smart Adherence Technology to Measure Lumacaftor/Ivacaftor Adherence in CF Subjects Homozygous for the F508del CFTR MutationNCT02823470results2018-09-27Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) TreatmentRandomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 4 | Terminatedsponsor's stated reason: Study terminated early due to slow recruitment of patients. | Drug: LUM/IVA · Device: activated smart device, de-activated smart device | Cystic Fibrosis | 24 | Aug 2017actual | — | 2018-09-27 |
| A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic FibrosisNCT02951182results2020-08-28Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Quadruple-masked · TreatmentStatistical Analysis Plan ↗ · Study Protocol ↗ | Phase 2 | Completed | Drug: TEZ, IVA, VX-440, Matched Placebo | Cystic Fibrosis | 74 | Aug 2017actual | — | 2020-08-28 |
| Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating MutationsNCT02971839results2020-08-26Change From Baseline in Sweat Chloride at Day 28Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Terminatedsponsor's stated reason: Decision by Sponsor. | Drug: VX-561, Placebo, IVA | Cystic Fibrosis | 11 | Aug 2017actual | — | 2020-08-26 |
| A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic FibrosisNCT03029455Safety and Tolerability assessments as determined by number of subjects with adverse events (AEs) and serious adverse events (SAEs)Randomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: VX-659, Tezacaftor, Ivacaftor, VX-659 Matching Placebo, Triple Combination (TC) Matching Placebos | Cystic Fibrosis | 163 | Aug 2017actual | — | 2017-09-05 |
| A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR MutationNCT02508207results2018-07-27Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: Tezacaftor/Ivacaftor, Ivacaftor, Tezacaftor/Ivacaftor matching placebo, Ivacaftor matching placebo | Cystic Fibrosis | 34 | Jun 2017actual | — | 2021-07-19 |
| A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) MutationNCT02392234results2018-06-12Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-661/Ivacaftor, Ivacaftor, Placebo matched to VX-661/ ivacaftor, Placebo matched to Ivacaftor | Cystic Fibrosis | 248 | Feb 2017actual | — | 2018-06-12 |
| A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With IvacaftorNCT02347657results2018-06-12Absolute Change From Baseline (Day 1) in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: VX-661 Plus Ivacaftor Combination, Ivacaftor, VX-661 Plus Ivacaftor Combination Placebo, Ivacaftor placebo | Cystic Fibrosis | 510 | 2017-01-20actual | — | 2018-06-12 |
| Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung DiseaseNCT02390219results2017-11-01Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)Open-label · Treatment | Phase 3 | Completed | Drug: Lumacaftor, Ivacaftor | Cystic Fibrosis, Advanced Lung Disease | 46 | Oct 2016actual | — | 2017-12-06 |
| A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR MutationNCT02514473results2017-10-23Absolute Change From Baseline in Lung Clearance Index 2.5 (LCI2.5) Through Week 24Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: VX-809, Placebo, VX-770 | Cystic Fibrosis | 206 | Sep 2016actual | — | 2017-10-23 |
| A Study Evaluating Safety and Pharmacokinetics of VX-440 in Combination With Tezacaftor/Ivacaftor in Healthy Adult SubjectsNCT03486236Safety and tolerability were based on the number and assessment of adverse events (AEs) and serious adverse events (SAEs)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-440, TEZ, IVA, Matched Placebos | Cystic Fibrosis | 16 | 2016-09-14actual | — | 2018-04-03 |
| A Study of the Efficacy and Safety of VX-150 in Subjects With Osteoarthritis of the KneeNCT02660424Change from baseline in Western Ontario and McMaster Universities (WOMAC) osteoarthritis index pain subscale score at Day 14Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: VX-150, Placebo | Osteoarthritis | 124 | Aug 2016actual | — | 2020-09-30 |
| A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR MutationNCT02516410results2018-06-12Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12Randomized · Triple-masked · Treatment | Phase 3 | Completed | Drug: VX-661 plus ivacaftor combination, Ivacaftor, Placebo (matched to VX-661 plus ivacaftor combination), Placebo (matched to ivacaftor) | Cystic Fibrosis | 168 | 2016-06-07actual | — | 2018-06-12 |
| An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic FibrosisNCT01863238observationalCataracts (lens opacities) | — | Completed | Other: Ophthalmologic examinations · Drug: Ivacaftor Exposed | Cystic Fibrosis | 95 | May 2016actual | — | 2016-08-23 |
| Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label ExpansionNCT02070744results2018-04-13PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to Ivacaftor | Cystic Fibrosis | 40 | 2016-05-27actual | — | 2025-09-24 |
| Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR MutationNCT01707290results2017-05-12Number of Participants With Treatment Emergent Adverse Events (TEAEs) or Serious Adverse Events (SAEs) in Ivacaftor ArmNon-randomized · Open-label · Treatment | Phase 3 | Completed | Drug: Ivacaftor | Cystic Fibrosis | 125 | Apr 2016actual | — | 2017-05-12 |
| A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic FibrosisNCT01931839results2017-05-12Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Randomized · Triple-masked · Treatment | Phase 3 | Completed | Drug: Lumacaftor Plus Ivacaftor Combination, Ivacaftor | Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation | 1,164 | Apr 2016actual | — | 2017-05-12 |
| Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating MutationNCT01946412results2017-02-01Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)Non-randomized · Open-label · Treatment | Phase 3 | Completed | Drug: Ivacaftor | Cystic Fibrosis | 33 | Dec 2015actual | — | 2017-02-01 |
| Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR MutationNCT01897233results2016-12-05Part A: Observed Plasma Concentration of Lumacaftor (LUM) and Ivacaftor (IVA) at Hour 4 Post-dose (C4h) on Day 1Open-label · Treatment | Phase 3 | Completed | Drug: Lumacaftor, Ivacaftor | Cystic Fibrosis | 62 | Oct 2015actual | — | 2017-06-20 |
| An Open-Label Study of the Effect of Telaprevir in Combination With Peginterferon Alfa-2b and Ribavirin in Pediatric Subjects Infected With Hepatitis C VirusNCT01701063results2016-07-20Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 1/2 | Terminated | Drug: Telaprevir, Peginterferon alfa-2b, Ribavirin | Hepatitis C | 42 | Apr 2015actual | — | 2016-07-20 |
| 24-week Study With Open Label Extension of VX-509, an Oral JAK3 Inhibitor, in Subjects Taking MethotrexateNCT01590459Proportion of subjects who achieve a 20% improvement in disease severity according to the American College of Rheumatology criteria, assessed using the C-reactive protein level (ACR20-CRP) responseRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: VX-509, VX-509 matching placebo | Rheumatoid Arthritis | 359 | Jul 2014actual | — | 2015-11-20 |
| A Phase 2/3 Open-label Extension Study to Evaluate Long-Term Safety and Efficacy With VX-509 in Subjects With Rheumatoid ArthritisNCT01830985Long-term safety and tolerability of VX-509 treatmentOpen-label · Treatment | Phase 2/3 | Completed | Drug: VX-509 | Rheumatoid Arthritis | 39 | Jul 2014actual | — | 2015-11-20 |
| Rollover Study of VX-770 in Cystic Fibrosis SubjectsNCT01117012results2015-07-07Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)Open-label · Treatment | Phase 3 | Completed | Drug: Ivacaftor | Cystic Fibrosis | 192 | May 2014actual | — | 2015-07-07 |
| Telaprevir, Peg-IFN-alfa-2a, and RBV in Treatment-Experienced Black/African American and Non-Black/African American Subjects With Genotype 1 Chronic Hepatitis CNCT01467492results2015-06-16Percentage of Participants With Sustained Viral Response 12 Weeks After Last Actual Dose of Study Drug (SVR12)Non-randomized · Open-label · Treatment | Phase 4 | Terminatedsponsor's stated reason: It was decided by Sponsor on 13 January 2014 to terminate study early at primary efficacy endpoint as part of a decision to modify drug development plan. | Drug: Telaprevir, Ribavirin · Biological: Pegylated Interferon Alfa-2a | Hepatitis C | 121 | May 2014actual | — | 2015-08-03 |
| A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-135 and Daclatasvir in Subjects With Genotype 1 Chronic Hepatitis C Chronic Hepatitis CNCT01842451The safety and tolerability as assessed by adverse events (AEs), vital signs, 12-lead electrocardiograms (ECGs), echocardiograms, and laboratory assessmentsRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: VX-135, Daclatasvir | Chronic Hepatitis C, CHC, HCV, Hepatitis C | 23 | May 2014actual | — | 2015-12-29 |
| Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR MutationNCT01225211results2015-10-05Cohort 1: Safety and Tolerability Based on Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: Lumacaftor, Ivacaftor, Lumacaftor Placebo, Ivacaftor Placebo | Cystic Fibrosis | 312 | Apr 2014actual | — | 2015-10-05 |
| An Open Label Study of the Effect of Telaprevir in Combination With Ribavirin and Peginterferon on HCV Infection in Stable Liver Transplant PatientsNCT01467505results2015-06-18Percentage of Participants With Sustained Viral Response 12 Weeks After Last Planned Dose of Study Drug (SVR12)Non-randomized · Open-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: The study was terminated early by the sponsor on 13 January 2014 due to a decision to modify the drug development plan. | Drug: Telaprevir, Ribavirin, Pegylated Interferon Alfa-2a, Immunosuppressant Regimen | Hepatitis C | 61 | Apr 2014actual | — | 2015-06-18 |
| Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR FunctionNCT01685801results2015-05-19Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of TreatmentRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: Ivacaftor, Placebo-matched-to-ivacaftor tablet | Cystic Fibrosis | 24 | Apr 2014actual | — | 2015-05-19 |
| A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR MutationNCT01807923results2015-08-31Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24Randomized · Double-masked · Treatment | Phase 3 | Completed | Drug: Lumacaftor Plus Ivacaftor Combination, Ivacaftor, Placebo | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | 559 | Apr 2014actual | — | 2015-08-31 |
| A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR MutationNCT01807949results2015-09-01Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24Randomized · Double-masked · Treatment | Phase 3 | Completed | Drug: Placebo, Lumacaftor Plus Ivacaftor Combination, Ivacaftor | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | 563 | Apr 2014actual | — | 2016-09-27 |
| Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) MutationNCT01531673results2018-04-13Safety as Determined by Adverse Events (AEs)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to ivacaftor | Cystic Fibrosis | 194 | Mar 2014actual | — | 2018-04-13 |
| Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating MutationNCT01705145results2015-04-24Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEsOpen-label · Treatment | Phase 3 | Completed | Drug: Ivacaftor | Cystic Fibrosis | 35 | Mar 2014actual | — | 2016-04-05 |
| Phase 1, QT/QTC Interval Study in Healthy SubjectsNCT01910415(Part A) Safety and tolerability of lumacaftor as measured by standard 12-lead ECGs, adverse events (AEs), vital signs, spirometry, and clinically significant laboratory assessmentsRandomized · Double-masked · Treatment | Phase 1 | Completed | Drug: Lumacaftor, Lumacaftor Placebo, Ivacaftor, Ivacaftor Placebo, moxifloxacin hydrochloride | Cystic Fibrosis | 200 | Mar 2014actual | — | 2014-08-11 |
| A Study to Treat Subjects With Telaprevir, Ribavirin, and Peginterferon Who Are Coinfected With HIV and Hepatitis C Virus (HCV)NCT01467479results2015-03-17Percentage of Participants With Sustained Viral Response 12 Weeks After Last Planned Dose of Study Drug (SVR12)Non-randomized · Open-label · Treatment | Phase 3 | Terminatedsponsor's stated reason: It was decided by Sponsor on 13 January 2014 to terminate study early at primary efficacy endpoint as part of a decision to modify drug development plan. | Drug: Telaprevir, Ribavirin, Highly Active Antiretroviral Therapy (HAART) · Biological: Pegylated Interferon Alfa-2a | Hepatitis C | 185 | Feb 2014actual | — | 2015-03-17 |
| A Phase 2 Study to Evaluate the Safety and Efficacy of VX-135 With Ribavirin in Treatment-Naïve Subjects With Chronic Hepatitis CNCT01726946The safety and tolerability as assessed by adverse events, vital signs, 12-lead electrocardiograms, echocardiograms (Cohorts 1 and 2 only), and laboratory assessmentsRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: VX-135, ribavirin | Chronic Hepatitis C | 10 | Feb 2014actual | — | 2015-04-16 |
| An Open-Label, Phase 1 Study in Healthy Adult Subjects to Examine the Effects of Multiple-Dose Ciprofloxacin on Ivacaftor and VX-661 in Combination With IvacaftorNCT02015507PK parameters of ivacaftor and metabolites following concomitant dosing with ciprofloxacin relative to ivacaftor administered aloneNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: ivacaftor, VX-661, ciprofloxacin | Cystic Fibrosis | 34 | Feb 2014actual | — | 2014-03-26 |
| Efficacy of a 12-Week Regimen of Telaprevir, Pegylated Interferon, and Ribavirin in Treatment-Naive and Prior Relapser Subjects With Interleukin28B (IL28B) CC GenotypeNCT01459913results2015-02-11Percentage of Subjects With Sustained Viral Response 12 Weeks After Last Planned Dose of Study Drug (SVR12)Randomized · Open-label · Treatment | Phase 3 | Terminatedsponsor's stated reason: The study was terminated early by the sponsor on 13 January 2014 due to a decision to modify the drug development plan. | Drug: Telaprevir, Pegylated Interferon Alfa-2a, Ribavirin | Hepatitis C, Chronic | 239 | Jan 2014actual | — | 2015-06-10 |
| A Magnetic Resonance Imaging Study and Arthroscopic Biopsy Substudy in Subjects With Active Rheumatoid Arthritis Receiving VX-509, an Oral JAK3 InhibitorNCT01754935Proportion of subjects achieving a ≥20% improvement in disease severity according to the American College of Rheumatology criteria (ACR20), using C reactive protein (CRP) (ACR20 CRP)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: VX-509, VX-509 matching placebo | Rheumatoid Arthritis | 43 | Jan 2014actual | — | 2015-05-19 |
| Virology Follow up Study in Subjects Previously Treated With TelaprevirNCT00916474observationalProportion of subjects who maintain undetectable HCV RNA over time after achieving an SVR following telaprevir-based treatment | — | Completed | Drug: telaprevir | Hepatitis C | 408 | Dec 2013actual | — | 2014-02-27 |
| Study of Lumacaftor in Combination With Ivacaftor in Subjects With Moderate Hepatic Impairment and Healthy SubjectsNCT01888393Lumacaftor pharmacokinetic parameters, including Cmax, tmax, AUC, Vd, CL/FNon-randomized · Open-label | Phase 1 | Completed | Drug: lumacaftor 200 mg q12h + ivacaftor 250 mg q12h | Hepatic Impairment, Healthy | 23 | Nov 2013actual | — | 2013-11-19 |
| A Safety and Efficacy Study of the Combination of VX-222 and Telaprevir in Treatment-Naïve Subjects With Genotype 1 Chronic Hepatitis C Virus InfectionNCT01080222Safety and TolerabilityRandomized · Open-label · Treatment | Phase 2 | Terminatedsponsor's stated reason: Study discontinued | Drug: telaprevir, VX-222, ribavirin, VX-222 · Biological: peginterferon-alfa-2a | Chronic Hepatitis C Virus Infection | 152 | Oct 2013actual | — | 2020-09-30 |
| Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)NCT01614457results2015-02-12Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Ivacaftor, Placebo | Cystic Fibrosis | 70 | Oct 2013actual | — | 2015-02-12 |
| Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating MutationNCT01614470results2014-10-29Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Ivacaftor, Placebo | Cystic Fibrosis | 39 | Oct 2013actual | — | 2014-10-29 |
| A Study to Evaluate the Efficacy and Safety of Quadruple Therapy (VX-222, Telaprevir,Peginterferon-Alfa-2a, Ribavirin) in Subjects With Chronic Hepatitis C With Compensated CirrhosisNCT01516918The proportion of subjects who have a sustained virologic response at 12 weeks after the last planned dose of treatment (SVR12)Open-label · Treatment | Phase 2 | Completed | Drug: VX-222, telaprevir, ribavirin · Biological: peginterferon-alfa-2a | Chronic Hepatitis C Virus | 103 | Sep 2013actual | — | 2014-10-20 |
| A Phase 1 Study to Examine the Drug-Drug Interaction of Ciprofloxacin, Itraconazole, and Rifampin on the Combination of Lumacaftor With Ivacaftor in Healthy Adult SubjectsNCT01768663Cohorts 1-3: PK parameters including Cmax, and AUC from time of dosing to time tau of lumacaftor and ivacaftor in the absence and presence of ciprofloxacin, itraconazole, and rifampinNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Lumacaftor, Ivacaftor, Ciprofloxacin, Itraconazole, Rifampin | Cystic Fibrosis | 80 | Sep 2013actual | — | 2013-11-15 |
| A Study to Evaluate the Efficacy and Safety of VX-765 in Subjects With Treatment-Resistant Partial EpilepsyNCT01501383Percent reduction in weekly seizure frequency during the Part A Late Treatment Period compared to the Part A Baseline PeriodRandomized · Triple-masked · Treatment | Phase 2 | Terminatedsponsor's stated reason: Terminated by Sponsor | Drug: VX-765 Part A, Placebo, VX-765 Part B | Epilepsy | 55 | Aug 2013actual | — | 2020-09-30 |
| Phase 1 PK Interaction Study Between VX-509 and Prednisone or Methylprednisolone in Healthy Male SubjectsNCT01886209PK parameters including Cmax, area under the concentration-time curve from the time of dosing extrapolated to infinity (AUC0 ∞), and AUC0-last for prednisone and its metabolite prednisolone with or without VX-509Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Prednisone, VX-509, Methylprednisolone | Drug Interactions | 28 | Aug 2013actual | — | 2013-08-14 |
| A Phase 1 Study to Investigate the Food Effect of Lumacaftor in Combination With IvacaftorNCT01899105Pharmacokinetic parameters of lumacaftor and ivacaftor, including Cmax, AUC0-tlast, and AUC0-∞Randomized · Open-label | Phase 1 | Completed | Drug: lumacaftor, ivacaftor | Cystic Fibrosis | 28 | Aug 2013actual | — | 2014-04-03 |
| VX-222 + Telaprevir + Ribavirin for 12 or 16 Weeks in Treatment-Naive Subjects With Genotype 1a Hepatitis CNCT01581138The proportion of subjects who have a sustained viral response (SVR) at 12 weeks after the last planned dose of treatmentRandomized · Open-label · Treatment | Phase 2 | Completed | Drug: VX-222, telaprevir, ribavirin | Chronic Hepatitis C Virus | 64 | Jun 2013actual | — | 2014-07-04 |
| Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D MutationNCT01161537results2014-07-31Part A: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Day 43Non-randomized · Single-masked · Treatment | Phase 2 | Completed | Drug: VX-770, Placebo | Cystic Fibrosis | 13 | Feb 2013actual | — | 2014-07-31 |
| A Study to Investigate VX-787 Given to Adult Volunteers Inoculated With Live Influenza VirusNCT01561807The primary endpoint is viral AUC as calculated in cell culture of nasal swabs (quantitation of nasal swabs for viral infectivity by cell culture), from initiation of VX-787 treatmentRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: VX-787, Placebo | Influenza Virus | 140 | Oct 2012actual | — | 2016-09-02 |
| Pharmacokinetics of Multiple Ascending Doses of VCH-222 in Subjects With Chronic Hepatitis C InfectionNCT00911963To assess the antiviral activity of VCH-222, in subjects with genotype 1 hepatitis C virus (HCV) infection after once (QD) or twice (b.i.d.) daily dosing for 3 days (Part A)Randomized · Quadruple-masked · Treatment | Phase 1/2 | Completed | Drug: VCH-222 or matching placebo, VCH-222 or matching placebo, ribavirin · Biological: peginterferon alfa-2a | Hepatitis C | 49 | Sep 2012actual | — | 2014-03-10 |
| A Study to Assess the Relative Bioavailability Three New Formulations of Telaprevir in Healthy SubjectsNCT01511432PK parameters: Maximum plasma concentration (Cmax), area under the concentration versus time curve (AUC) from time 0 to infinity (AUC0-∞)Randomized · Open-label | Phase 1 | Completed | Drug: telaprevir formulation A, telaprevir Formulation B, telaprevir Formulation C, telaprevir Formulation D | Chronic Hepatitis C | 64 | Jun 2012actual | — | 2012-07-04 |
| Safety and Efficacy of Telaprevir in Combination With Peginterferon Alfa-2a and Ribavirin in Subjects Co-Infected With Hepatitis C Virus (HCV) and HIVNCT00983853results2013-10-10Proportion of Subjects Achieving Undetectable HCV RNA at Week 12Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: telaprevir or matching placebo, telaprevir or matching placebo, ribavirin (fixed dose), ribavirin (weight-based dose) · Biological: peginterferon alfa-2a | Hepatitis C, HIV Infections | 62 | Mar 2012actual | — | 2013-10-10 |
| Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D MutationNCT01262352results2013-01-30Absolute Change From Baseline in Lung Clearance Index (LCI)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Ivacaftor, Placebo | Cystic Fibrosis | 21 | Nov 2011actual | — | 2013-02-11 |
| A 12-week Study of 4 Doses of VX-509 in Subjects With Active Rheumatoid ArthritisNCT01052194Proportion of subjects who achieve an ACR20 responseRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: Placebo, VX-509, VX-509, VX-509, VX-509 | Rheumatoid Arthritis | 206 | Jul 2011actual | — | 2012-12-13 |
| Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy SubjectsNCT01216046PK parameters (including concentration, exposure and half-life) of VX-809 and its metabolite in plasma in the presence and absence of VX 770Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-809, VX-770, VX-809 placebo, VX-770 placebo | Cystic Fibrosis | 48 | May 2011actual | — | 2012-01-18 |
| Drug-Drug Interaction Study Between Telaprevir and BuprenorphineNCT01275599Blood levels of buprenorphineNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: telaprevir, buprenorphine/naloxone | Hepatitis C, Opioid-Related Disorders | 16 | Apr 2011actual | — | 2011-06-08 |
| Study of VX-770 in Subjects With Moderate Hepatic Impairment and in Matched Healthy SubjectsNCT01208285VX-770 pharmacokinetic parametersNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-770 | In Development for Cystic Fibrosis | 24 | Dec 2010actual | — | 2011-01-12 |
| Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D MutationNCT00909727results2012-08-21Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Ivacaftor, Placebo | Cystic Fibrosis | 52 | Nov 2010actual | — | 2012-08-21 |
| Study of VX-765 in Subjects With Treatment-resistant Partial EpilepsyNCT01048255Safety and tolerability (vital sign assessments, physical and neurological examinations, laboratory assessments, and adverse events)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: VX-765 | Partial Epilepsy | 60 | Nov 2010actual | — | 2014-01-27 |
| Study of VX-985 in Subjects With Chronic Hepatitis CNCT01144936Safety and tolerability (vital signs, 12-lead electrocardiograms, physical examinations, laboratory assessments, and adverse events)Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-985 or matching placebo, VX-985 or matching placebo, VX-985 or matching placebo | Chronic Hepatitis C | 21 | Oct 2010actual | — | 2011-03-09 |
| Study of VX-770 on DesipramineNCT01153542VX-770 and Desipramine pharmacokinetic parametersNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-770, VX-770 | In Development for Cystic Fibrosis | 24 | Aug 2010actual | — | 2010-12-09 |
| Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D MutationNCT00909532results2012-08-21Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24Randomized · Quadruple-masked · Treatment | Phase 3 | Completed | Drug: Ivacaftor, Placebo | Cystic Fibrosis | 167 | Jul 2010actual | — | 2013-01-18 |
| Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR MutationNCT00953706results2012-08-21Part A : Absolute Change From Part A Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 16Randomized · Quadruple-masked · Treatment | Phase 2 | Terminatedsponsor's stated reason: Following review of results obtained from a pre-specified 6-month analysis of Part B data the study was terminated on the basis of futility. | Drug: Ivacaftor, Placebo | Cystic Fibrosis | 140 | Jul 2010actual | — | 2015-09-11 |
| A Study Evaluating 24-Week and 48-Week Telaprevir-Based Regimen in Treatment Naïve Subjects With Genotype 1 Chronic Hepatitis C Who Achieve an Extended Rapid Viral ResponseNCT00758043results2011-07-21Proportion of Randomized Subjects Achieving Sustained Viral Response (SVR), Demonstrated by Achieving Undetectable HCV RNA 24 Weeks After Last Dose of Study Treatment (SVR24)Randomized · Open-label · Treatment | Phase 3 | Completed | Drug: telaprevir, ribavirin · Biological: peginterferon alfa-2a | Hepatitis C | 540 | Jun 2010actual | — | 2021-03-26 |
| A Phase 3 Study of Telaprevir in Combination With Pegasys® and Copegus® in Treatment-Naive Subjects With Genotype 1 Hepatitis C Virus (HCV)NCT00627926results2011-07-21Number of Subjects Achieving Sustained Viral Response (SVR), Demonstrated by Achieving Undetectable Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) Levels 24 Weeks After Last Planned Dose of Study TreatmentRandomized · Quadruple-masked · Treatment | Phase 3 | Completed | Biological: Pegylated Interferon Alfa 2a · Drug: Telaprevir, Ribavirin · Other: Placebo | Hepatitis C | 1,095 | May 2010actual | — | 2014-08-08 |
| A Study to Examine the Effects of Telaprevir on the Pharmacokinetics of Cyclosporine and Tacrolimus in Healthy AdultsNCT01038167Part A only: Pharmacokinetic parameters of cyclosporine (Cmax, AUC0-tlast, AUC0-inf, tmax, t1/2)Non-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: telaprevir, telaprevir, cyclosporine, cyclosporine, tacrolimus, tacrolimus | Hepatitis C | 20 | Apr 2010actual | — | 2010-04-08 |
| A Phase I Trial of AVN944, an IMPDH Inhibitor, in Adults With Advanced Stage Solid TumorsNCT009237281. The dose limiting toxicities with AVN944 in patients with advanced stage solid tumors; 2. Maximum tolerated dose with AVN944 in patients with advanced stage solid tumorsNon-randomized · Open-label · Treatment | Not applicable | Withdrawn | Drug: AVN944 | Refractory Solid Tumors | 0 | Mar 2010 | — | 2014-01-10 |
| Study of VX-770 and Rifampin in Healthy Male SubjectsNCT01018368VX 770 pharmacokinetic (PK) parametersNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-770, Rifampin | Cystic Fibrosis | 24 | Mar 2010actual | — | 2010-05-10 |
| Study of VX-770 on Midazolam and Rosiglitazone and the Effect of Fluconazole on VX-770NCT01060566Midazolam, Rosiglitazone and VX 770 pharmacokinetic (PK) parameterNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: VX-770, VX-770 | Cystic Fibrosis | 24 | Mar 2010actual | — | 2010-04-16 |
| A Rollover Study for Subjects Participating in the Control Arm of Study VX06-950-106, VX05-950-104 and VX05-950-104EU Whose Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) Levels Did Not Respond to TherapyNCT00535847results2011-07-25Percentage of Subjects With Undetectable Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Week 24 After the Completion of TreatmentNon-randomized · Open-label · Treatment | Phase 2 | Completed | Drug: Telaprevir, Ribavirin, Pegylated interferon alfa 2a | Hepatitis C | 117 | Feb 2010actual | — | 2014-08-05 |
| Drug-Drug Interaction Study of VCH-222 and Telaprevir in Healthy SubjectsNCT00958152Safety and tolerability based on vital signs, 12-lead electrocardiograms (ECGs), physical examination, laboratory assessments, and adverse eventsRandomized · Quadruple-masked | Phase 1 | Completed | Drug: VCH-222, VCH-222, VCH-222, telaprevir | Hepatitis C | 30 | Jan 2010actual | — | 2010-01-07 |
| AVN944 in Combination With Gemcitabine for the Treatment of Pancreatic CancerNCT00493441SafetyNon-randomized · Open-label · Treatment | Phase 2 | Terminated | Drug: AVN944 | Pancreatic Cancer | 28 | Dec 2009actual | — | 2020-09-30 |
| Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene MutationNCT00865904results2015-08-28Safety and Tolerability Based on Adverse Events (AEs)Randomized · Triple-masked · Treatment | Phase 2 | Completed | Drug: VX-809, Placebo | Cystic Fibrosis | 93 | Dec 2009actual | — | 2015-08-28 |
| Drug-Drug Interaction Study of VX-809 and VX-770 in Healthy SubjectsNCT00966602Pharmacokinetic (PK) parameters of VX-770 and its metabolites in plasma in the presence and absence of VX-809Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VX-809, VX-770, VX-809 & VX-770, Placebo | Cystic Fibrosis | 24 | Dec 2009actual | — | 2010-01-05 |
| A Study of Telaprevir (VX-950), Pegasys and Copegus in Hepatitis C (PROVE3)NCT00420784results2011-07-21Percentage of Subjects With Undetectable Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Week 24 After the Completion of Study Drug DosingRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Telaprevir, Ribavirin, Pegylated Interferon Alfa 2a, Matching Placebo | Hepatitis C | 465 | Dec 2008actual | — | 2014-08-05 |
| A Safety Study for Cethrin (BA-210) in the Treatment of Acute Thoracic and Cervical Spinal Cord InjuriesNCT00500812The primary goal of this study is to determine the safety and tolerability of Cethrin when administered in conjunction with fibrin sealant to the dura mater of the spinal cord.Non-randomized · Open-label · Treatment | Phase 1/2 | Completed | Drug: Cethrin | Spinal Cord Injury | 48 | Dec 2008actual | — | 2016-04-14 |
| Safety,Tolerability and Pharmacokinetics of Multiple Ascending Doses of VCH 916 in Subjects With Chronic Hep C InfectionNCT00623649The primary objective of this trial is to assess the antiviral activity, safety, and tolerability of VCH-916 monotherapy in adult subjects with chronic HCV-infection.Randomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: VCH 916, Placebo | HCV Infection | 42 | Oct 2008actual | — | 2014-04-04 |
| Safety Study of Ivacaftor in Subjects With Cystic FibrosisNCT00457821results2012-10-05Number of Subjects With Adverse Events (Combined Part 1 and Part 2)Randomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Ivacaftor 25 mg/75 mg, Ivacaftor 75 mg/150 mg, Ivacaftor 150 mg or 250 mg, Placebo | Cystic Fibrosis | 39 | Aug 2008actual | — | 2012-10-05 |
| Study of Telaprevir in Subjects With Hepatic ImpairmentNCT00509210PharmacokineticsNon-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: telaprevir (VX-950) | Hepatic Insufficiency | — | Jun 2008actual | — | 2008-10-29 |
| Phase 2 Study of VX-950, Pegasys® With and Without Copegus® in Hepatitis CNCT00372385results2011-07-21Percentage of Subjects With Undetectable Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Week 24 After the Completion of Study Drug DosingRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Ribavirin, Pegylated Interferon Alfa 2a, Placebo, Telaprevir | Chronic Hepatitis C | 334 | May 2008actual | — | 2014-07-23 |
| Phase 2 Study of VX-950, Pegasys®, and Copegus® in Hepatitis CNCT00336479results2011-07-21Percentage of Subjects With Undetectable Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Week 24 After the Completion of Study Drug DosingRandomized · Quadruple-masked · Treatment | Phase 2 | Completed | Drug: Telaprevir, Ribavirin, Pegylated Interferon Alfa 2a · Other: Placebo | Chronic Hepatitis C | 263 | Feb 2008actual | — | 2014-07-23 |
| VX-710, Doxorubicin, and Vincristine for the Treatment of Patients With Recurrent Small Cell Lung CancerNCT00003847Response RateNon-randomized · Open-label · Treatment | Phase 2 | Terminated | Drug: biricodar dicitrate, doxorubicin hydrochloride, vincristine sulfate | Lung Cancer | 36 | Feb 2001actual | — | 2013-09-05 |
| Merimepodib (MMPD) in Triple Combination for the Treatment of Chronic Hepatitis CNCT00088504Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: Merimepodib, PEG-Interferon-alpha 2a (Pegasys®), Ribavirin (Copegus®) | Hepatitis C, Hepatitis | 315 | — | — | 2007-12-21 |
| Phase 2 Clinical Study in Psoriasis With Oral Investigational Drug VX-765NCT00205465Incidence of adverse eventsRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: VX-765 | Psoriasis | 64 | — | — | 2007-12-05 |
| Phase 2 Clinical Study in Rheumatoid Arthritis With an Investigational Oral p38 MAP Kinase Inhibitor VX-702NCT00205478The primary endpoint for the study is the ACR20 response at Week 12.Randomized · Double-masked · Treatment | Phase 2 | Completed | Drug: VX-702 | Rheumatoid Arthritis | 300 | — | — | 2007-12-07 |
| VX-950 and Peginterferon for Hepatitis CNCT00251199Viral kineticsRandomized · Double-masked | Phase 1 | Completed | Drug: VX-950, peginterferon | Hepatitis C | 20 | — | — | 2007-12-21 |
| Phase 2 Study of VX-950, Pegasys and Copegus in Hepatitis CNCT00262483Safety of triple combination therapy.Non-randomized · Open-label | Phase 2 | Completed | Drug: VX-950, ribavirin, peginterferon alfa-2a | Hepatitis C | 12 | — | — | 2007-12-21 |
| Trial of AVN-944 in Patients With Advanced Hematologic MalignanciesNCT00273936Non-randomized · Open-label | Phase 1 | Completed | Drug: AVN-944 capsules for oral administration | Acute Leukemia, Chronic Leukemia, Multiple Myeloma, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Waldenstrom's Macroglobulinemia | 72 | — | — | 2011-08-04 |
| A Proof-of-concept Study of VCH-759 for the Treatment of Hepatitis C-infection.NCT00389298Change in baseline HCV plasma RNA (i.e., viral load) at Day 11.Randomized · Double-masked · Treatment | Phase 1/2 | Completed | Drug: VCH-759 (BCH-27759) | Hepatitis C, Chronic | 36 | — | — | 2009-09-24 |
| A Study in Rheumatoid Arthritis With an Investigational Oral p38 MAP Kinase Inhibitor VX-702NCT00395577To evaluate the effects of VX-702 administered with concomitant methotrexate (MTX), and MTX aloneRandomized · Double-masked · Treatment | Phase 2 | Completed | Drug: VX-702 | Rheumatoid Arthritis | 120 | — | — | 2007-12-07 |
| Single and Multiple Dose Escalation Study of VX-509 in Healthy SubjectsNCT00789126Safety and Tolerability as measured by adverse events (AEs) and clinically relevant changes in laboratory values, vital signs, electrocardiograms (ECGs), and physical examination variablesRandomized · Double-masked | Phase 1 | Completed | Drug: VX-509 | Healthy | 72 | — | — | 2009-02-19 |
| VX-770 Expanded Access ProgramNCT01381289expanded access | — | Approved for marketing | Drug: VX-770 | Cystic Fibrosis | — | — | — | 2012-02-09 |
| Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic FibrosisNCT03278314expanded access | — | Approved for marketing | Drug: tezacaftor/ivacaftor | Cystic Fibrosis | — | — | — | 2018-11-02 |
| VX-445/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients Heterozygous for F508del Mutation and a Minimal Function Mutation (F/MF Genotypes)NCT04058210expanded access | — | Approved for marketing | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | — | — | — | 2019-11-21 |
| ELX/TEZ/IVA Expanded Access Program for Cystic Fibrosis (CF) Patients With at Least One F508del MutationNCT04702360expanded access | — | Approved for marketing | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | — | — | — | 2021-07-15 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19