Cystic Fibrosis trial readouts
registry pull 2026-08-19Primary-completion dates that listed drug and biotech companies filed with ClinicalTrials.gov for studies in Cystic Fibrosis. They are the sponsor's own estimated windows, revised as a study runs — not announcement dates. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Upcoming readout windows
next 180 days · by month3 studies across 2 companies, in the next 180 days.
October 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| VRTX | Vertex Pharmaceuticals Incorporated | 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) | Phase 3 | Active, not recruiting | 2026-10-30 | in 71 d | 2026-07-21 |
November 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| VRTX | Vertex Pharmaceuticals Incorporated | 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) | Phase 1 | Recruiting | 2026-11-19 | in 91 d | 2026-07-16 |
December 2026 1
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| KRYS | Krystal Biotech, Inc. | A Study Assessing KB407 for the Treatment of Cystic FibrosisNCT05504837To evaluate safety and tolerability of KB407 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in physical examinations, vital signs, ECG, and clinical laboratory test results | Phase 1 | Recruiting | Dec 2026 | ≤ 133 d | 2026-05-07 |
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.
Every registered study in Cystic Fibrosis
most recently updated first193 studies match. Showing the first 100 of 193 studies — page through, or narrow the filters.
| Ticker | Name | Market cap | Study | Phase | Status | Interventions | Conditions | Enrollment | Start | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Active, not recruiting | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 297 | 2022-11-23 | 2027-04-06 | in 229 d | 2026-08-11 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 1 | Completed | Drug: VX-828, Placebo, Itraconazole, Midazolam, Tezacaftor, VX-118, Placebo, Deutivacaftor, VX-828 | Cystic Fibrosis | 165 | 2023-12-12 | 2026-07-10actual | — | 2026-08-10 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Active, not recruiting | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 822 | 2022-11-08 | 2026-10-30 | in 71 d | 2026-07-21 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 1 | Recruiting | Drug: VX-581, Placebo, D-IVA, TEZ | Cystic Fibrosis | 136 | 2025-12-09 | 2026-11-19 | in 91 d | 2026-07-16 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Active, not recruiting | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 50 | 2024-11-21 | 2027-09-30 | in 406 d | 2026-06-05 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Metabolites | Phase 3 | Active, not recruiting | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 210 | 2022-06-21 | 2030-06-30 | in 1,410 d | 2026-05-28 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 1/2 | Active, not recruiting | Drug: VX-522 mRNA therapy, IVA | Cystic Fibrosis | 26 | 2023-02-27 | 2026-04-21actual | — | 2026-05-22 |
| ARCT | Arcturus Therapeutics, Inc. | $233M | Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic FibrosisNCT06747858Incidence, severity and dose relationship of adverse events | Phase 2 | Recruiting | Biological: ARCT-032 | Cystic Fibrosis, CFTR Gene Mutation | 33 | 2024-12-12 | Jun 2027 | ≤ 314 d | 2026-05-08 |
| KRYS | Krystal Biotech, Inc. | $9.9B | A Study Assessing KB407 for the Treatment of Cystic FibrosisNCT05504837To evaluate safety and tolerability of KB407 based upon assessment of adverse events (frequency, severity, relatedness), and changes from baseline in physical examinations, vital signs, ECG, and clinical laboratory test results | Phase 1 | Recruiting | Biological: KB407 (Nebulization) | Cystic Fibrosis | 20 | 2023-06-30 | Dec 2026 | ≤ 133 d | 2026-05-07 |
| SION | Sionna Therapeutics Inc. | $269M | Proof-of-concept Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of SION-719 When Added to TrikaftaNCT07108153To evaluate the incidence of treatment emergent adverse events [Safety and Tolerability] when SION-719 is administered to people with cystic fibrosis (CF) who are taking a standard stable dose of physician-prescribed Trikafta | Phase 2 | Active, not recruiting | Drug: SION-719, Placebo-to-match SION-719 | Cystic Fibrosis (CF) | 16 | 2025-11-03 | Jul 2026 | — | 2026-05-06 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Enrolling by invitation | Drug: VNZ/TEZ/D-IVA | Cystic Fibrosis | 174 | 2023-08-11 | 2029-07-30 | in 1,075 d | 2026-04-29 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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-IVA | Phase 1 | Completed | Drug: VNZ/TEZ/D-IVA, Rosuvastatin | Cystic Fibrosis | 18 | 2026-01-12 | 2026-02-28actual | — | 2026-04-02 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 1 | Recruiting | Drug: VX-272, Placebo | Cystic Fibrosis | 128 | 2026-02-25 | 2027-03-23 | in 215 d | 2026-03-31 |
| INSM | Insmed Incorporated | $28.1B | A Study to Assess the Safety, Tolerability, and Pharmacokinetics of Brensocatib Tablets in Adults With Cystic FibrosisNCT05090904results2026-03-12Maximum Plasma Concentration (Cmax) of Brensocatib on Day 1 | Phase 2 | Completed | Drug: Brensocatib, Placebo | Cystic Fibrosis | 29 | 2021-11-30 | 2022-11-01actual | — | 2026-03-12 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 71 | 2022-01-17 | 2026-01-29actual | — | 2026-03-03 |
| SNY | Sanofi | — | Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic FibrosisNCT03375047results2026-02-09Parts A, B and D: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events | Phase 1/2 | Completed | Drug: MRT5005, Normal saline | Cystic Fibrosis | 42 | 2018-05-10 | 2022-03-15actual | — | 2026-02-09 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 | 2024-07-09 | 2025-11-27actual | — | 2025-12-31 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Metabolites | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 70 | 2023-06-27 | 2025-09-04actual | — | 2025-10-06 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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-IVA | Phase 1 | Completed | Drug: VNZ/TEZ/D-IVA, VNZ/TEZ/D-IVA | Cystic Fibrosis | 34 | 2024-03-13 | 2024-05-23actual | — | 2025-09-26 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 2 | Completed | Drug: VX-661, Ivacaftor, Placebo matched to VX-661, Placebo matched to Ivacaftor | Cystic Fibrosis | 40 | Mar 2014 | 2016-05-27actual | — | 2025-09-24 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 507 | 2018-10-09 | 2023-01-09actual | — | 2025-09-24 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 12 | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 82 | 2021-10-12 | 2022-07-26actual | — | 2025-09-24 |
| SION | Sionna Therapeutics Inc. | $269M | Safety, Tolerability, and Pharmacokinetics of Multiple Dose Combinations of SION-451 and Complementary Modulators SION-2222 and SION-109 in Healthy Participants.NCT07035990To evaluate the incidence of treatment emergent adverse events [Safety and Tolerability] of SION-451 and SION-109 when administered in combination to healthy participants | Phase 1 | Recruiting | Drug: SION-451, SION-2222, SION-109, Placebo SION-451, Placebo SION-2222, Placebo SION-109 | Cystic Fibrosis (CF) | 144 | 2025-07-23 | Jun 2026 | — | 2025-09-03 |
| PHGE | BiomX Ltd | $4M | Study With Phage for CF Subjects With Pseudomonas Lung InfectionNCT06998043Change in sputum Pseudomonas aeruginosa (PsA) burden at 8 weeks (EOT) | Phase 2 | Recruiting | Biological: BX004 · Other: Placebo | Chronic Pseudomonas Aeruginosa Infection, Cystic Fibrosis (CF) | 63 | 2025-07-02 | Mar 2026 | — | 2025-07-22 |
| PHGE | BiomX, Inc. | $4M | Nebulized Bacteriophage Therapy in Cystic Fibrosis Patients With Chronic Pseudomonas Aeruginosa Pulmonary InfectionNCT05010577Safety and tolerability | Phase 1/2 | Completed | Drug: BX004-A, Placebo | Chronic Pseudomonas Aeruginosa Infection, Cystic Fibrosis | 43 | 2022-06-21 | 2024-03-10actual | — | 2025-07-18 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Plasma | Phase 1 | Completed | Drug: VX-828, VX-828 | Cystic Fibrosis | 68 | 2025-04-01 | 2025-05-05actual | — | 2025-06-04 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 64 | 2020-02-17 | 2024-02-24actual | — | 2025-05-18 |
| AMGN | Amgen | $229.9B | Safety, Tolerability and Efficacy of MP-376 Given for 28 Days to Cystic Fibrosis (CF) PatientsNCT00677365results2012-06-12Change in P. Aeruginosa Density | Phase 2 | Completed | Drug: MP-376, Placebo | Cystic Fibrosis (CF) | 151 | Jun 2008 | Jun 2009actual | — | 2024-12-27 |
| AMGN | Amgen | $229.9B | MP-376 (Aeroquin™, Levofloxacin for Inhalation) in Patients With Cystic FibrosisNCT01180634results2024-04-30Time to an Exacerbation | Phase 3 | Completed | Drug: Aeroquin, Placebo | Cystic Fibrosis | 330 | 2010-11-04 | 2012-05-07actual | — | 2024-12-18 |
| AMGN | Amgen | $229.9B | Trial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) PatientsNCT01270347results2024-05-21Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 3 | Completed | Drug: MP-376 (Levofloxacin Solution for Inhalation), TIS (Tobramycin Inhalation Solution) | Cystic Fibrosis | 282 | Jan 2011 | Oct 2012actual | — | 2024-12-13 |
| AMGN | Amgen | $229.9B | Safety, Pharmacokinetic and Pharmacodynamic Study of MP-376 in Patients With Cystic FibrosisNCT00503490Safety and tolerability of MP-376 administered twice a day for 14 days | Phase 1 | Completed | Drug: MP-376 (Levofloxacin solution for Inhalation), placebo | Cystic Fibrosis | 40 | Jun 2007 | Dec 2007actual | — | 2024-12-04 |
| AMGN | Amgen | $229.9B | "Safety, Tolerability and Pharmacokinetics of MP-376 Administered for 14 Days to Stable Pediatric (CF) Patients"NCT00840333Safety and Tolerability of MP-376 administered for 14 days to CF patients ages 6-16 | Phase 1 | Completed | Drug: MP-376 (Levofloxacin solution for Inhalation) | Cystic Fibrosis | 27 | Apr 2009 | Dec 2009actual | — | 2024-12-04 |
| ARCT | Arcturus Therapeutics, Inc. | $233M | Safety, Tolerability, and Pharmacokinetics of ARCT-032 in Healthy Adult Subjects and Adults With Cystic Fibrosis.NCT05712538Incidence, severity and dose-relationship of AEs | Phase 1 | Completed | Drug: ARCT-032 · Other: Placebo | Cystic Fibrosis | 39 | 2023-02-15 | 2024-07-29actual | — | 2024-11-13 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 TEAEs | Phase 3 | Completed | Drug: IVA | Cystic Fibrosis | 86 | 2017-08-16 | 2023-10-02actual | — | 2024-10-23 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | 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 | 2021-09-14 | 2023-05-12actual | — | 2024-10-03 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: LUM/IVA | Cystic Fibrosis | 52 | 2020-02-24 | 2023-08-22actual | — | 2024-09-19 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA · Other: Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) | Cystic Fibrosis | 307 | 2022-05-09 | 2023-07-05actual | — | 2024-08-01 |
| ABBV | AbbVie | $457.5B | Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With CreonNCT05069597results2024-07-23Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85 | Phase 4 | Completed | Drug: CREON | Cystic Fibrosis, Chronic Pancreatitis | 30 | 2021-10-14 | 2023-07-07actual | — | 2024-07-23 |
| ABBV | AbbVie | $457.5B | Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic FibrosisNCT04853368results2024-07-16Cohorts 1 and 2: Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | Phase 2 | Terminatedsponsor's stated reason: Strategic considerations | Drug: ABBV-576, Galicaftor, Placebo, Navocaftor, ABBV-119 | Cystic Fibrosis (CF) | 48 | 2021-09-20 | 2023-06-05actual | — | 2024-07-16 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | 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 | 2021-10-27 | 2023-05-18actual | — | 2024-06-13 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | A Study to Evaluate the Relative Bioavailability and Food Effect of a VX-118 Tablet FormulationNCT06312787Maximum Observed Plasma Concentration (Cmax) of VX-118 | Phase 1 | Completed | Drug: VX-118 | Cystic Fibrosis | 15 | 2024-04-02 | 2024-05-04actual | — | 2024-06-03 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Method | Phase 1 | Completed | Drug: VNZ/TEZ/D-IVA | Cystic Fibrosis | 10 | 2024-03-26 | 2024-04-16actual | — | 2024-05-16 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 120 | 2021-01-11 | 2023-03-24actual | — | 2024-05-08 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: TEZ/IVA, IVA | Cystic Fibrosis | 130 | 2018-04-25 | 2020-10-28actual | — | 2024-04-19 |
| INSM | Insmed Incorporated | $28.1B | Single Dose Escalation Study of TR02 (Sustained Lipid Inhalation Technology [SLIT™] Amikacin) in Participants With Cystic Fibrosis (CF) Having Chronic Infections of Pseudomonas AeruginosaNCT06238856Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE) | Phase 1 | Completed | Drug: SLIT™ Amikacin, Placebo | Cystic Fibrosis | 18 | 2004-05-12 | 2005-02-08actual | — | 2024-02-02 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 251 | 2019-12-05 | 2022-12-16actual | — | 2024-01-16 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: TEZ/IVA, IVA | Cystic Fibrosis | 1,131 | Aug 2015 | May 2019actual | — | 2023-09-28 |
| ABBV | AbbVie | $457.5B | A Study to Compare US Marketed Creon Manufactured With a Modernized Process at an Alternate Manufacturing Site and Manufactured With the Approved Manufacturing Process at an Alternate Active Pharmaceutical Ingredient Site, in Participants With Exocrine Pancreatic Insufficiency Due to Cystic FibrosisNCT03924947results2023-09-28Part 1 Coefficient of Fat Absorption (CFA) | Phase 4 | Completed | Drug: Pancrelipase, Pancrelipase | Cystic Fibrosis | 36 | 2019-10-23 | 2022-07-11actual | — | 2023-09-28 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: IVA | Cystic Fibrosis | 57 | 2016-06-02 | 2022-06-28actual | — | 2023-09-07 |
| KRYS | Krystal Biotech, Inc. | $9.9B | A Study of Inhaled KB407 for the Treatment of Cystic FibrosisNCT05095246To evaluate the safety and tolerability of KB407 in subjects with Cystic Fibrosis through adverse events as assessed by NCI-CTCAE v5.0 | Phase 1 | Withdrawnsponsor's stated reason: Pandemic, logistical, and recruitment challenges | Biological: KB407 (Nebulization) | Cystic Fibrosis | 0 | 2022-03-08 | 2024-07-01 | — | 2023-08-14 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 48 | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 69 | 2021-01-15 | 2022-07-14actual | — | 2023-08-03 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 172 | 2020-05-04 | 2022-12-21actual | — | 2023-07-28 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 1 | Completed | Drug: Vanzacaftor, Vanzacaftor Placebo, Moxifloxacin, Moxifloxacin Placebo | Cystic Fibrosis | 56 | 2023-04-26 | 2023-06-14actual | — | 2023-07-14 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 86 | 2022-01-14 | 2022-12-20actual | — | 2023-07-11 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 458 | 2019-08-09 | 2022-12-14actual | — | 2023-07-06 |
| ABBV | AbbVie | $457.5B | A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222NCT03969888results2023-06-28Absolute Change From Baseline Through Day 29 in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | Phase 2 | Completed | Drug: ABBV-3067, Placebo ABBV-3067, ABBV-2222, Placebo ABBV-2222 | Cystic Fibrosis | 78 | 2019-12-11 | 2022-06-09actual | — | 2023-06-28 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Metabolites | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 83 | 2020-11-19 | 2022-06-03actual | — | 2023-06-28 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 2 | Completed | Drug: VX-121, TEZ, VX-561, TEZ/IVA, IVA, Placebo | Cystic Fibrosis | 87 | 2019-04-30 | 2019-12-10actual | — | 2023-04-20 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Metabolites | Phase 1 | Completed | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 16 | 2022-07-22 | 2023-03-16actual | — | 2023-03-30 |
| CRBP | Corbus Pharmaceuticals Inc. | $195M | Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic FibrosisNCT03451045results2023-01-18Pulmonary Exacerbation (PEx) Rate Over 28 Weeks | Phase 2 | Completed | Drug: Lenabasum 20 mg, Lenabasum 5 mg · Other: Placebo | Cystic Fibrosis | 447 | 2017-12-22 | 2020-06-17actual | — | 2023-01-18 |
| SVRA | Savara Inc. | $1.1B | Trial of Inhaled Molgramostim in Cystic Fibrosis Subjects With Nontuberculous Mycobacterial InfectionNCT03597347results2021-10-11Number of Participants With Sputum NTM Culture Conversion to Negative | Phase 2 | Terminatedsponsor's stated reason: Recruitment for trial stopped early due to limitations from COVID-19, additionally changes in the standard of care for CF patients during the study period resulted in confounding of the primary endpoint. | Drug: Molgramostim nebulizer solution · Device: PARI eFlow nebulizer system | Mycobacterium Infections, Nontuberculous, Cystic Fibrosis (CF) | 14 | 2019-06-20 | 2020-10-02actual | — | 2023-01-10 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 IVA | Phase 3 | Completed | Drug: LUM, IVA | Cystic Fibrosis | 61 | 2018-09-07 | 2021-10-29actual | — | 2023-01-06 |
| SVRA | Savara Inc. | $1.1B | AeroVanc in the Treatment of Methicillin-resistant Staphylococcus Aureus Infection in Patients With Cystic FibrosisNCT03181932results2021-11-05Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Percent Predicted | Phase 3 | Completed | Drug: Vancomycin inhalation powder, Placebo inhalation powder, Vancomycin inhalation powder | MRSA, Cystic Fibrosis | 188 | 2017-09-20 | 2020-07-28actual | — | 2022-12-16 |
| XAIR | Beyond Air Inc. | $5M | A Pilot Study to Assess the Effect of Intermittent iNO on the Treatment of NTM Lung Infection in CF and Non-CF PatientsNCT04685720Treatment-Emergent SAEs | Not applicable | Completed | Device: LungFit | Non-Tuberculous Mycobacterial Pneumonia, Cystic Fibrosis, Mycobacterial Pneumonia, Mycobacterium Abscessus Infection, Mycobacterium Avium Complex | 15 | 2020-12-07 | 2022-07-28actual | — | 2022-12-16 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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-IVA | Phase 1 | Completed | Drug: VX-121/TEZ/D-IVA | Cystic Fibrosis | 16 | 2022-09-15 | 2022-11-09actual | — | 2022-12-12 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 48 | Phase 2 | Completed | Drug: LUM/IVA, LUM/IVA, Placebo | Cystic Fibrosis | 51 | 2018-08-10 | 2020-10-09actual | — | 2022-11-02 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA · Other: Placebo (matched to ELX/TEZ/IVA), Placebo (matched to IVA) | Cystic Fibrosis | 121 | 2020-06-19 | 2021-05-17actual | — | 2022-07-26 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | 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 | 2018-03-20 | 2019-05-03actual | — | 2022-07-14 |
| GILD | Gilead Sciences | $177.9B | Study of Aztreonam for Inhalation in Children With Cystic Fibrosis and New Infection of the Airways by Pseudomonas Aeruginosa BacteriaNCT03219164results2022-05-16Percentage of Participants With Pseudomonas Aeruginosa (PA)-Negative Cultures Through 28 Days Post-Treatment in the 14-Day Treatment Group vs 28-Day Treatment Group | Phase 3 | Terminatedsponsor's stated reason: Study was terminated early by sponsor due to the challenges and risks introduced by Coronavirus disease 2019 (COVID-19) pandemic. | Drug: AZLI, Placebo | Pseudomonas Aeruginosa Respiratory Tract Infection/Colonization, Cystic Fibrosis | 149 | 2017-11-28 | 2020-05-27actual | — | 2022-06-14 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Terminatedsponsor's stated reason: At Sponsor's Discretion | Drug: VX-659/TEZ/IVA, IVA | Cystic Fibrosis | 484 | 2018-07-13 | 2020-09-09actual | — | 2022-01-25 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 2 | Completed | Drug: VX-561, IVA, Placebo, Placebo | Cystic Fibrosis | 77 | 2019-04-17 | 2020-08-20actual | — | 2022-01-25 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 | 2021-06-30 | 2021-12-20actual | — | 2022-01-19 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 1/2 | Completed | Drug: IVA, TEZ/IVA, VX-445, Matched Placebo, TEZ, VX-561, VX-445 | Cystic Fibrosis | 225 | 2017-01-23 | 2018-03-27actual | — | 2022-01-18 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 IVA | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA | Cystic Fibrosis | 71 | 2018-10-02 | 2020-08-07actual | — | 2021-10-22 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Score | Phase 3 | Completed | Drug: ELX/TEZ/IVA, TEZ/IVA, IVA | Cystic Fibrosis | 176 | 2019-10-03 | 2020-07-24actual | — | 2021-08-18 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 28 | Phase 2 | Completed | Drug: Tezacaftor/Ivacaftor, Ivacaftor, Tezacaftor/Ivacaftor matching placebo, Ivacaftor matching placebo | Cystic Fibrosis | 34 | Feb 2016 | Jun 2017actual | — | 2021-07-19 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Group | Phase 3 | Completed | Drug: ELX/TEZ/IVA, IVA, TEZ/IVA | Cystic Fibrosis | 271 | 2019-08-28 | 2020-06-12actual | — | 2021-07-02 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: LUM/IVA, LUM/IVA | Cystic Fibrosis | 246 | Aug 2015 | Aug 2018actual | — | 2021-05-24 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | 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 | 2017-08-08 | 2018-02-28actual | — | 2021-04-22 |
| IONS | Ionis Pharmaceuticals, Inc. | $9.9B | A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic FibrosisNCT03647228Safety and Tolerability as Measured by the Number of Participants with at least one Treatment-Emergent Adverse Event | Phase 1 | Completed | Drug: IONIS-ENaCRx, Placebo | Healthy Subjects, Cystic Fibrosis | 98 | 2018-12-13 | 2020-10-13actual | — | 2021-02-04 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 2 | Completed | Drug: VX-152, TEZ/IVA, IVA, Placebo, Placebo | Cystic Fibrosis | 80 | Nov 2016 | Jan 2018actual | — | 2021-01-28 |
| ABBV | AbbVie | $457.5B | Prevalence of Antibodies to Selected Porcine Viruses in Patients With Cystic Fibrosis Receiving Porcine-derived Pancreatic Enzyme Replacement TherapyNCT01858519observationalSeroprevalence of antibodies to hepatitis E virus (HEV) and other selected porcine viruses | — | Completed | — | Cystic Fibrosis | 1,310 | Aug 2013 | Sep 2016actual | — | 2020-12-09 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 | Apr 2015 | Oct 2020actual | — | 2020-11-13 |
| PTCT | PTC Therapeutics | $6.1B | Extension Study of Ataluren (PTC124) in Cystic FibrosisNCT01140451results2020-10-19Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | Phase 3 | Completed | Drug: Ataluren | Cystic Fibrosis | 191 | 2010-08-12 | 2013-12-02actual | — | 2020-10-19 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 2 | Completed | Drug: TEZ, IVA, VX-440, Matched Placebo | Cystic Fibrosis | 74 | Oct 2016 | Aug 2017actual | — | 2020-08-28 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 28 | Phase 2 | Terminatedsponsor's stated reason: Decision by Sponsor. | Drug: VX-561, Placebo, IVA | Cystic Fibrosis | 11 | Dec 2016 | Aug 2017actual | — | 2020-08-26 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: LUM/IVA | Cystic Fibrosis | 57 | 2017-05-12 | 2019-07-17actual | — | 2020-08-07 |
| INSM | Insmed Incorporated | $28.1B | Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™)NCT00777296results2019-07-16Clinically Significant Laboratory Abnormalities. | Phase 1/2 | Completed | Drug: ARIKACE™, Placebo | Cystic Fibrosis | 66 | 2007-02-22 | 2008-02-27actual | — | 2020-07-30 |
| INSM | Insmed Incorporated | $28.1B | Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension PhaseNCT03905642results2019-05-14Adverse Event Profile of 560 mg Once Daily Dose of Arikayce™ Administered for Six Cycles Over Eighteen Months. | Phase 2 | Completed | Drug: Arikayce™ | Cystic Fibrosis | 49 | 2009-01-08 | 2010-11-02actual | — | 2020-07-30 |
| INSM | Insmed Incorporated | $28.1B | Extension Study of Liposomal Amikacin for Inhalation in Cystic Fibrosis (CF) Patients With Chronic Pseudomonas Aeruginosa (Pa) InfectionNCT01316276results2019-07-26Treatment Emergent Adverse Events (TEAEs) up to Day 672 | Phase 3 | Completed | Drug: Liposomal amikacin for inhalation | Cystic Fibrosis | 206 | 2012-10-05 | 2015-07-16actual | — | 2020-06-17 |
| PTCT | PTC Therapeutics | $6.1B | Safety and Efficacy of Ataluren (PTC124) for Cystic FibrosisNCT00237380Change From Baseline to End of Treatment in Total Chloride Transport | Phase 2 | Completed | Drug: Ataluren | Cystic Fibrosis | 24 | 2005-11-30 | 2006-05-31actual | — | 2020-06-11 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: VX-445/TEZ/IVA, IVA, Placebo | Cystic Fibrosis | 405 | 2018-06-15 | 2019-04-24actual | — | 2020-05-19 |
| PTCT | PTC Therapeutics | $6.1B | Study of Ataluren (PTC124™) in Cystic FibrosisNCT00803205results2020-05-14Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline | Phase 3 | Completed | Drug: Ataluren, Placebo | Cystic Fibrosis | 238 | 2009-09-08 | 2011-11-12actual | — | 2020-05-14 |
| PTCT | PTC Therapeutics | $6.1B | Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)NCT02139306results2020-05-14Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48 | Phase 3 | Completed | Drug: Ataluren (PTC124®), Placebo | Cystic Fibrosis | 279 | Aug 2014 | Nov 2016actual | — | 2020-05-14 |
| PTCT | PTC Therapeutics | $6.1B | Extension Study of Ataluren in Participants With Nonsense Mutation Cystic FibrosisNCT02456103results2020-04-15Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 3 | Terminatedsponsor's stated reason: Cystic Fibrosis (CF) data from the double-blind CF Study PTC124-GD-021-CF did not meet endpoints. | Drug: Ataluren | Cystic Fibrosis | 246 | 2015-08-31 | 2017-06-02actual | — | 2020-04-27 |
| PTCT | PTC Therapeutics | $6.1B | Study of Ataluren (PTC124) in Cystic FibrosisNCT02107859results2020-03-25Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Phase 3 | Terminatedsponsor's stated reason: Based on the results of study PTC124-GD-021-CF (NCT02139306), clinical development of ataluren in cystic fibrosis was discontinued and this study was closed. | Drug: Ataluren | Cystic Fibrosis | 61 | 2014-05-23 | 2017-06-05actual | — | 2020-03-25 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Completed | Drug: VX-659/TEZ/IVA, IVA, Placebo | Cystic Fibrosis | 385 | 2018-03-07 | 2019-02-05actual | — | 2020-03-13 |
| PTCT | PTC Therapeutics | $6.1B | A Study of Ataluren in Pediatric Participants With Cystic FibrosisNCT00458341results2020-03-06Change From Baseline in Total Chloride Transport at Day 14 of Cycles 1 and 2 | Phase 2 | Completed | Drug: Ataluren | Cystic Fibrosis | 30 | 2007-03-23 | 2008-02-29actual | — | 2020-03-06 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 IVA | Phase 3 | Completed | Drug: TEZ, TEZ/IVA, IVA | Cystic Fibrosis | 83 | Nov 2016 | Sep 2018actual | — | 2020-03-04 |
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.
How Cystic Fibrosis is matched — 4 exact registry spellings
A study is on this page when its ClinicalTrials.gov record names one of these conditions exactly (letter case aside). Nothing is matched by substring or stem, so a longer condition that merely contains one of these words is a different page. The list was curated by hand and last measured 2026-08-19; a spelling filed by only one study was not curated, so the counts here are a floor rather than the registry's own total for the disease.
- Cystic Fibrosis
- Cystic Fibrosis (CF)
- Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
- In Development for Cystic Fibrosis
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