Sickle Cell Disease trial readouts
registry pull 2026-08-19Primary-completion dates that listed drug and biotech companies filed with ClinicalTrials.gov for studies in Sickle Cell Disease. 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 month2 studies across 2 companies, in the next 180 days.
January 2027 2
| Ticker | Name | Study | Phase | Status | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|
| BMY | Bristol-Myers Squibb | A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell DiseaseNCT06481306Number of participants with adverse events (AEs) | Phase 1/2 | Recruiting | 2027-01-06 | in 139 d | 2026-08-18 |
| SNY | Sanofi | Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell DiseaseNCT07247188Number of participants reporting immediate adverse events (AEs) | Phase 3 | Recruiting | 2027-01-07 | in 140 d | 2026-04-15 |
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.
Every registered study in Sickle Cell Disease
most recently updated first73 studies match.
| Ticker | Name | Market cap | Study | Phase | Status | Interventions | Conditions | Enrollment | Start | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| IRON | Disc Medicine, Inc | $3.1B | A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)NCT07187973Safety and tolerability of DISC-3405 administration in participants with SCD | Phase 1 | Recruiting | Drug: DISC-3405 | Sickle Cell Disease | 24 | 2026-01-06 | Jun 2027 | ≤ 314 d | 2026-08-19 |
| PFE | Pfizer | $155.3B | A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCDNCT05431088Part A | Phase 2/3 | Recruiting | Drug: Osivelotor | Sickle Cell Disease | 389 | 2022-09-22 | 2030-12-30 | in 1,593 d | 2026-08-18 |
| BMY | Bristol-Myers Squibb | $134.9B | A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell DiseaseNCT06481306Number of participants with adverse events (AEs) | Phase 1/2 | Recruiting | Drug: BMS-986470, Placebo, Famotidine, Pantoprazole | Anemia, Sickle Cell, Healthy Volunteers | 224 | 2024-07-17 | 2027-01-06 | in 139 d | 2026-08-18 |
| NVO | Novo Nordisk A/S | — | A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or ThalassaemiaNCT06609226Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately | Phase 3 | Recruiting | Drug: Etavopivat A, Etavopivat B, Etavopivat C | Sickle Cell Disease, Thalassemia | 480 | 2025-01-10 | 2030-12-30 | in 1,593 d | 2026-08-13 |
| NVO | Novo Nordisk A/S | — | A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell DiseaseNCT06612268Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact | Phase 3 | Recruiting | Drug: Etavopivat, Placebo | Sickle Cell Disease | 408 | 2025-02-17 | 2027-08-27 | in 372 d | 2026-08-13 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat StudyNCT05777993Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study | Phase 4 | Enrolling by invitation | Drug: Mitapivat | Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia | 59 | 2023-07-06 | Aug 2029 | ≤ 1,107 d | 2026-08-12 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | A Long-term Follow-up Study in Participants Who Received CTX001NCT04208529New malignancies | Phase 3 | Enrolling by invitation | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia | 160 | 2021-01-20 | 2039-09-30 | in 4,789 d | 2026-08-11 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)NCT07656415Percentage of Subjects who are Transfusion Free From Week 4 Through Week 52 | Phase 3 | Recruiting | Drug: Mitapivat Matched Placebo, Mitapivat | Sickle Cell Disease | 159 | 2026-07-21 | Aug 2029 | ≤ 1,107 d | 2026-08-04 |
| SNY | Sanofi | — | The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell DiseaseNCT06975865Annualized rate of clinical VOC | Phase 3 | Recruiting | Drug: Rilzabrutinib, Placebo | Sickle Cell Disease | 192 | 2025-08-12 | 2027-07-23 | in 337 d | 2026-07-29 |
| NVO | Novo Nordisk A/S | — | Research Study Investigating How Well NDec Works in People With Sickle Cell DiseaseNCT05405114Change in total haemoglobin | Phase 2 | Completed | Drug: NDec - oral decitabine-tetrahydrouridine, HU - Hydroxyurea, Placebo | Sickle Cell Disease | 96 | 2022-07-07 | 2025-07-24actual | — | 2026-07-22 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 3 | Active, not recruiting | Biological: CTX001 | Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases | 13 | 2022-05-02 | 2027-06-06 | in 290 d | 2026-07-01 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)NCT05031780Phase 2: Percentage of Participants With Hemoglobin (Hb) Response | Phase 2/3 | Active, not recruiting | Drug: Mitapivat · Other: Mitapivat-matching placebo, Mitapivat-matching placebo | Sickle Cell Disease | 286 | 2022-02-11 | 2025-10-30actual | — | 2026-06-23 |
| FULC | Fulcrum Therapeutics | $295M | Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell DiseaseNCT07431398Plasma concentration of pociredir under fasted and fed conditions | Phase 1 | Terminatedsponsor's stated reason: Sponsor decision | Drug: Pociredir | Sickle Cell Disease | 13 | 2025-12-13 | 2026-06-01actual | — | 2026-06-18 |
| PFE | Pfizer | $155.3B | Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell DiseaseNCT04255875Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs | Phase 1 | Completed | Biological: Placebo, PF-07209326, PF-07209326 | Healthy, Sickle Cell Anemia | 52 | 2020-02-05 | 2023-07-07actual | — | 2026-06-15 |
| PFE | Pfizer | $155.3B | A Study to Learn About Sickle Cell Disease In Adult PatientsNCT05407805results2026-06-10observationalPhysician-reported Medical Utilization (MU) Vaso-occlusive Crisis (VOC) Rate | — | Completed | Other: Electronic Diary | Sickle Cell Disease | 98 | 2022-02-10 | 2024-06-24actual | — | 2026-06-10 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)NCT06924970Percentage of Participants With Hb Response | Phase 2 | Active, not recruiting | Drug: Tebapivat, Tebapivat Matched Placebo | Sickle Cell Disease | 59 | 2025-05-01 | May 2026 | — | 2026-05-29 |
| SNY | Sanofi | — | Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell DiseaseNCT07247188Number of participants reporting immediate adverse events (AEs) | Phase 3 | Recruiting | Biological: PCV21, 20vPCV | Sickle Cell Disease | 100 | 2026-01-20 | 2027-01-07 | in 140 d | 2026-04-15 |
| FULC | Fulcrum Therapeutics | $295M | Open-Label Extension Study to Pioneer Study 6058-SCD-101NCT07401823Number of participants reporting Treatment Emergent Adverse Events (TEAEs) | Phase 2 | Enrolling by invitation | Drug: Pociredir | Sickle Cell Disease | 50 | 2026-03-30 | 2030-02-04 | in 1,264 d | 2026-04-14 |
| CERS | Cerus Corporation | $556M | Biotin-Acridine Red Cell Exchange KineticsNCT07155031Recovery | Phase 2 | Withdrawnsponsor's stated reason: This study has been suspended due to a lack of funding. The protocol will be reconsidered within the next 6-12 months if/as funding becomes available. | Device: Pathogen reduced red blood cells | Sickle Cell Disease | 0 | 2025-10-15 | 2026-09-30 | in 41 d | 2026-04-09 |
| NVO | Novo Nordisk A/S | — | Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell DiseaseNCT05725902Effect of etavopivat on cerebral blood flow (CBF) | Phase 2 | Completed | Drug: Etavopivat | Sickle Cell Disease | 12 | 2024-03-09 | 2026-03-09actual | — | 2026-04-08 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study of Mitapivat in Participants With Sickle Cell Disease and NephropathyNCT06286046Percentage of Participants With Albumin Creatinine Ratio (ACR) Response | Phase 2 | Withdrawnsponsor's stated reason: Withdrawn prior to study start per sponsor decision. No patients were enrolled. | Drug: Mitapivat | Sickle Cell Disease, Nephropathy | 0 | Mar 2026 | May 2027 | ≤ 284 d | 2026-04-03 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Months | Phase 3 | Withdrawnsponsor's stated reason: Modification to the clinical development plan | Biological: Exa-cel | Sickle Cell Disease | 0 | 2027-07-31 | 2033-12-31 | in 2,690 d | 2026-03-25 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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 Time | Phase 3 | Recruiting | Biological: CTX001 | Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia | 26 | 2022-08-02 | 2027-06-09 | in 293 d | 2026-03-23 |
| PFE | Pfizer | $155.3B | GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)NCT05632354results2026-03-19Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Phase 2/3 | Terminatedsponsor's stated reason: Study terminated as data obtained from this non-placebo-controlled trial may not be informative for the interpretation of the benefits and risks of osivelotor | Drug: Osivelotor | Sickle Cell Disease | 47 | 2023-01-05 | 2025-02-13actual | — | 2026-03-19 |
| PFE | Pfizer | $155.3B | Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell DiseaseNCT04218084results2025-06-13Change From Baseline in Time-Averaged Maximum of Mean Velocity (TAMMV) Arterial Cerebral Blood Flow at Week 24 | Phase 3 | Terminatedsponsor's stated reason: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment. | Drug: Voxelotor, Placebo | Sickle Cell Disease | 236 | 2020-11-11 | 2024-01-18actual | — | 2026-03-10 |
| FULC | Fulcrum Therapeutics | $295M | Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PociredirNCT05169580Treatment-Emergent Adverse Events | Phase 1 | Completed | Drug: Pociredir oral capsule(s) | Sickle Cell Disease, Sickle Cell Anemia | 45 | 2021-12-13 | 2026-01-20actual | — | 2026-02-12 |
| BEAM | Beam Therapeutics Inc. | $2.7B | A Long-term Follow-up Study in Patients Who Received BEAM-101NCT07373639observationalLong-Term Safety Monitoring and Mortality Assessment | — | Recruiting | Drug: Long-Term Follow-up Study of patients who received BEAM-101 | Hemoglobinopathy, Sickle Cell Disease | 50 | 2025-12-15 | 2043-02-12 | in 6,020 d | 2026-01-28 |
| NVO | Novo Nordisk A/S | — | A Study on the Effect of Etavopivat on Heart Rhythm in Healthy ParticipantsNCT07023029Part A: Number of treatment-emergent adverse events (AEs) | Phase 1 | Completed | Drug: Etavopivat, Moxifloxacin, Placebo | Healthy Volunteers, Sickle Cell Disease, Thalassemia | 33 | 2025-06-09 | 2025-09-12actual | — | 2026-01-02 |
| PFE | Pfizer | $155.3B | Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCDNCT04930445results2025-12-31observationalChange in Hemoglobin From Pre-Oxbryta Treatment (Baseline) Through Month 60 Post-Oxbryta | — | Terminatedsponsor's stated reason: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research, and requires further assessment. | Drug: Oxbryta® (voxelotor) 500mg Tablets | Sickle Cell Disease | 265 | 2022-02-04 | 2024-10-10actual | — | 2025-12-31 |
| NVO | Novo Nordisk A/S | — | A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese ParticipantsNCT06581627AUC0-inf,etavopivat: Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose | Phase 1 | Completed | Drug: Etavopivat | Healthy Volunteers, Sickle Cell Disease, Thalassemia | 24 | 2024-08-26 | 2024-10-31actual | — | 2025-12-29 |
| PFE | Pfizer | $155.3B | A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell DiseaseNCT05348915Incidence of treatment-emergent adverse events (TEAEs). | Phase 3 | Terminatedsponsor's stated reason: Sponsor decision to terminate study based on results of parent study GBT2104-131. | Drug: Inclacumab | Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease | 242 | 2022-03-29 | 2025-11-07actual | — | 2025-12-17 |
| BEAM | Beam Therapeutics Inc. | $2.7B | BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell DiseaseNCT05456880Change in annualized number of severe VOCs (Vascular-occlusive Crisis) relative to baseline | Phase 1/2 | Active, not recruiting | Biological: BEAM-101 | Sickle Cell Disease | 15 | 2022-08-30 | Feb 2028 | ≤ 558 d | 2025-12-16 |
| PFE | Pfizer | $155.3B | A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive CrisesNCT04935879results2025-12-02Rate of Vaso-occlusive Crises (VOCs) [Adjudicated] Through Week 48 | Phase 3 | Completed | Drug: Inclacumab, Placebo | Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis | 241 | 2021-10-04 | 2024-06-06actual | — | 2025-12-02 |
| PFE | Pfizer | $155.3B | Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031NCT03573882results2025-11-18Number of Participants With Sickle Cell Disease (SCD)-Related Treatment Emergent Adverse Events (TEAEs) | Phase 3 | Terminatedsponsor's stated reason: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment. | Drug: Voxelotor | Sickle Cell Disease | 179 | 2018-06-06 | 2024-11-12actual | — | 2025-11-18 |
| PFE | Pfizer | $155.3B | Open-Label Extension of VoxelotorNCT04188509results2025-11-13Number of Participants With Treatment Emergent Adverse Events (AEs) | Phase 3 | Terminatedsponsor's stated reason: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment. | Drug: Voxelotor | Sickle Cell Disease | 162 | 2019-11-18 | 2024-11-01actual | — | 2025-11-13 |
| NVO | Novo Nordisk A/S | — | A Research Study of the Effect of Food on Etavopivat in Healthy ParticipantsNCT06433661AUC0-inf, etavopivat: Area under the etavopivat plasma concentration-time curve from 0 hours and extrapolated to infinity after a single dose | Phase 1 | Completed | Drug: Etavopivat | Healthy Volunteers Sickle Cell Disease, Thalassemia | 16 | 2024-05-28 | 2024-07-06actual | — | 2025-10-10 |
| NVO | Novo Nordisk A/S | — | A Research Study of the Effect of Etavopivat on Other Drugs in Healthy ParticipantsNCT06813924Cmax, digoxin, SD: Maximum observed digoxin plasma concentration with and without etavopivat at steady state | Phase 1 | Completed | Drug: Etavopivat, Digoxin, Pitavastatin, Metformin, Midazolam, Rosuvastatin | Healthy Volunteers Sickle Cell Disease, Thalassemia | 37 | 2025-02-06 | 2025-05-27actual | — | 2025-09-25 |
| PFE | Pfizer | $155.3B | Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell DiseaseNCT02850406results2025-08-15Part A: Maximum Concentration (Cmax) of Voxelotor in Whole Blood | Phase 2 | Terminatedsponsor's stated reason: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment. | Drug: Voxelotor | Sickle Cell Disease | 147 | 2016-07-05 | 2023-10-02actual | — | 2025-08-15 |
| VRTX | Vertex Pharmaceuticals Incorporated | $133.9B | 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) | Phase 2/3 | Completed | Biological: CTX001 | Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies | 63 | 2018-11-27 | 2025-07-07actual | — | 2025-08-11 |
| PFE | Pfizer | $155.3B | Resolution of Sickle Cell Leg Ulcers With VoxelotorNCT05561140results2025-07-09Percentage of Participants Who Achieved Resolution of the Target Ulcers by Week 12 in Randomized Treatment Period | Phase 3 | Terminatedsponsor's stated reason: Emerging clinical data evaluated by Pfizer and shared with regulatory authorities indicates that the risk profile of voxelotor in people with SCD exceeds the benefits observed in previously generated global research and requires further assessment. | Drug: Voxelotor Oral Tablet · Other: Placebo | Sickle Cell Disease, Leg Ulcers | 88 | 2022-05-30 | 2024-06-26actual | — | 2025-07-09 |
| EDIT | Editas Medicine, Inc. | $442M | A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301NCT06363760observationalAdverse events of special interest (AESI) | — | Enrolling by invitation | Other: Safety and efficacy assessments | Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies | 54 | 2024-06-17 | Aug 2040 | ≤ 5,125 d | 2025-04-02 |
| EDIT | Editas Medicine, Inc. | $442M | A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)NCT04853576Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs) | Phase 1/2 | Active, not recruiting | Genetic: EDIT-301 | Sickle Cell Disease, Hemoglobinopathies | 45 | 2021-05-04 | Aug 2025 | — | 2025-01-31 |
| PFE | Pfizer | $155.3B | A GBT021601 ADME Microtracer Study in Healthy VolunteersNCT05718687results2025-01-08GBT021601 Concentration in Whole Blood and Plasma at Predose | Phase 1 | Completed | Drug: GBT021601 | Sickle Cell Disease | 9 | 2022-12-22 | 2023-08-10actual | — | 2025-01-08 |
| PFE | Pfizer | $155.3B | A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive CrisesNCT04927247results2024-12-11Percentage of Participants With at Least 1 VOC That Required Admission to a Healthcare Facility and Treatment With Parenteral Pain Medication Within 90 Days of Randomization | Phase 3 | Terminatedsponsor's stated reason: Study was terminated due to poor accrual and associated recrutiment challenges | Drug: Inclacumab, Placebo | Sickle Cell Disease, Vaso-occlusive Crisis, Vaso-occlusive Pain Episode in Sickle Cell Disease | 72 | 2021-12-09 | 2023-11-24actual | — | 2024-12-11 |
| PFE | Pfizer | $155.3B | Voxelotor CYP and Transporter Cocktail Interaction StudyNCT05981365results2024-11-22Part A: Maximum Observed Plasma Concentration (Cmax) for Bupropion | Phase 1 | Completed | Drug: Voxelotor, Bupropion, Repaglinide, Flurbiprofen, Omeprazole, Midazolam, Metformin, Furosemide, Rosuvastatin | Sickle Cell Disease | 44 | 2023-04-17 | 2023-10-04actual | — | 2024-11-22 |
| PFE | Pfizer | $155.3B | Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment OptionsNCT04724421expanded access | — | No longer available | Drug: Voxelotor | Sickle Cell Disease | — | — | — | — | 2024-11-20 |
| NVS | Novartis | — | Characteristics and Treatment Patterns of Patients With Sickle Cell Disease in Globin Research Network for Data and Discovery RegistryNCT06662812observationalNumber of Patients in the Globin Research Network for Data and Discovery (GRNDaD) Registry who Received a Disease Modifying Therapy (DMT) | — | Completed | — | Sickle Cell Disease | 1,665 | 2021-10-06 | 2023-12-01actual | — | 2024-10-29 |
| NVS | Novartis | — | Real World Patient Characteristics and Treatment Patterns From Crizanlizumab Use: Preliminary Analysis From Select Sickle Cell CentersNCT06662825observationalAge at Index Date | — | Completed | — | Sickle Cell Disease | 376 | 2022-12-13 | 2023-11-01actual | — | 2024-10-29 |
| PFE | Pfizer | $155.3B | Voxelotor Brain Oxygenation and Neurocognitive StudyNCT05228821Change in CBF | Phase 4 | Withdrawnsponsor's stated reason: The study has been stopped due to Sponsor's business reasons. The termination is not a result of any safety concerns or change in the benefit-risk ratio. | Drug: Voxelotor Oral Tablet | Sickle Cell Disease | 0 | 2023-08-09 | 2023-08-23actual | — | 2024-08-09 |
| PFE | Pfizer | $155.3B | A Study to Evaluate GBT021601-012 Single Dose and Multiple Dose in Participants With Sickle Cell Disease (SCD)NCT04983264results2024-06-10Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Phase 1 | Completed | Drug: GBT021601 | Sickle Cell Disease | 6 | 2021-05-21 | 2022-12-06actual | — | 2024-06-10 |
| PFE | Pfizer | $155.3B | Voxelotor Neurocognitive Function StudyNCT05228834results2024-05-02Change From Baseline in Executive Abilities Composite Score Assessed Using NIH Toolbox Cognition Module At Week 12 | Phase 3 | Terminatedsponsor's stated reason: Data will not inform further development of Voxelotor | Drug: Voxelotor Only Product in Oral Dose Form, Placebo | Sickle Cell Disease | 1 | 2022-06-24 | 2022-09-30actual | — | 2024-05-02 |
| AGIO | Agios Pharmaceuticals, Inc. | $2.1B | A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell DiseaseNCT04536792SAD and MAD: Number of Participants Who Experienced at Least One Adverse Event (AE), by Severity | Phase 1 | Completed | Drug: AG-946, AG-946 Matched Placebo | Healthy Volunteers, Anemia, Sickle Cell | 122 | 2020-07-10 | 2023-12-19actual | — | 2024-04-16 |
| PFE | Pfizer | $155.3B | Retrospective Real World Oxbryta® Data Collection and Analysis StudyNCT04930328observationalChange from pre-Oxbryta treatment period in Hemoglobin (Hb) | — | Completed | Drug: Oxbryta® (voxelotor) 500-mg Tablets | Sickle Cell Disease | 216 | 2021-03-20 | 2022-02-25actual | — | 2024-03-06 |
| PFE | Pfizer | $155.3B | Dose Escalation Study to Evaluate the Safety, Tolerability, PK and PD of Voxelotor in Patients With SCDNCT04247594results2023-11-21Treatment-emergent Adverse Events (AEs) | Phase 2 | Terminatedsponsor's stated reason: Data will not inform further development of Voxelotor | Drug: Voxelotor | Sickle Cell Disease | 6 | 2020-01-09 | 2021-06-08actual | — | 2023-11-21 |
| PFE | Pfizer | $155.3B | Actigraphy Improvement With Voxelotor (ActIVe) StudyNCT04400487results2023-11-21Change From Baseline in Total Daily Physical Activity (Counts Per Minute) to Week 10-12 | Phase 4 | Completed | Drug: Voxelotor | Sickle Cell Disease, Sickle Cell Anemia | 25 | 2020-12-21 | 2022-09-08actual | — | 2023-11-21 |
| PFE | Pfizer | $155.3B | Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)NCT03036813results2021-01-07Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24 | Phase 3 | Completed | Drug: voxelotor · Other: Placebo | Sickle Cell Disease | 449 | Dec 2016 | 2019-10-08actual | — | 2023-07-27 |
| PFE | Pfizer | $155.3B | A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy ParticipantsNCT05036512Safety, as assessed by frequency and severity of adverse events (AEs) | Phase 1 | Completed | Drug: GBT021601 | Sickle Cell Disease | 129 | 2020-12-09 | 2022-11-02actual | — | 2023-07-25 |
| CYCN | Cyclerion Therapeutics | $19M | A Study of the Effect of IW-1701 (Olinciguat), a Stimulator of Soluble Guanylate Cyclase (sGC), on Patients With Sickle Cell Disease (SCD)NCT03285178results2023-07-21Double-Blind Treatment: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | Phase 2 | Completed | Drug: IW-1701, Placebo | Sickle Cell Disease | 88 | 2017-12-22 | 2020-07-22actual | — | 2023-07-21 |
| PFE | Pfizer | $155.3B | Expanded Access to Voxelotor for Patients With Sickle Cell Disease Who Have No Alternative Treatment OptionsNCT03943615expanded access | — | No longer available | Drug: Voxelotor | Sickle Cell Disease | — | — | — | — | 2023-07-18 |
| FULC | Fulcrum Therapeutics | $295M | Safety, Tolerability and Pharmacokinetics of FTX-6058NCT04586985Treatment-Emergent Adverse Events | Phase 1 | Completed | Drug: FTX-6058/placebo oral capsule(s), FTX-6058/placebo oral capsule(s), FTX-6058 - Two Dosing Periods, FTX-6058 / Midazolam Syrup, FTX-6058/placebo oral capsule(s) | Healthy Adult Subjects, Sickle Cell Disease | 109 | 2020-10-26 | 2022-11-15actual | — | 2022-12-12 |
| AZN | AstraZeneca | — | PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4)NCT03492931Peak Plasma Concentration (Cmax) of Ticagrelor | Phase 1 | Completed | Drug: Ticagrelor | Sickle Cell Disease | 21 | 2018-03-28 | 2019-05-07actual | — | 2021-05-10 |
| AZN | AstraZeneca | — | Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell DiseaseNCT03615924results2021-04-09Number of Vaso-Occlusive Crisis Events | Phase 3 | Terminatedsponsor's stated reason: Recommendation from an independent data monitoring committee (DMC) and accepted by AstraZeneca. | Drug: Ticagrelor, Placebo | Sickle Cell Disease | 193 | 2018-09-26 | 2020-08-13actual | — | 2021-04-09 |
| BMRN | BioMarin Pharmaceutical | $13.0B | A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell DiseaseNCT00445978results2021-01-28Number of Subjects With Treatment Emergent Adverse Events (TEAEs) | Phase 2 | Completed | Drug: Sapropterin Dihydrochloride | Sickle Cell Disease | 32 | May 2007 | Aug 2008actual | — | 2021-02-25 |
| AZN | AstraZeneca | — | Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease.NCT04293172Number of Vaso-Occlusive Crises | Phase 3 | Withdrawnsponsor's stated reason: Study halted prematurely, prior to enrolment of first patient. | Drug: Brilinta, Placebo | Sickle Cell Disease | 0 | 2020-06-30 | 2022-10-10 | — | 2020-07-15 |
| NVS | Novartis | — | A 3-year, Prospective, Non-interventional, Multicenter Registry in Sickle Cell Disease (SCD) PatientsNCT01220115observationalDocument current treatment patterns, natural history and outcomes in patients with sickle cell disease | — | Completed | — | Sickle Cell Disease | 498 | Jan 2010 | Sep 2014actual | — | 2019-12-09 |
| INCY | Incyte Corporation | $24.7B | A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With Sickle Cell DiseaseNCT03132324Safety and tolerability of INCB059872 assessed by monitoring frequency, duration, and severity of adverse events | Phase 1 | Terminatedsponsor's stated reason: This study is terminated due to a business decision not to pursue INCB059782 in Sickle Cell Disease indication. | Drug: INCB059872 | Sickle Cell Disease | 12 | 2017-04-20 | 2018-10-03actual | — | 2019-10-28 |
| LLY | Eli Lilly and Company | $1.15T | A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)NCT01794000results2016-07-27Number of Vaso-Occlusive Crisis (VOC) Events Per Participant Per Year (Rate of VOC) | Phase 3 | Terminatedsponsor's stated reason: The study is being terminated for lack of efficacy. | Drug: Prasugrel, Placebo | Sickle Cell Disease | 341 | Apr 2013 | Jul 2015actual | — | 2019-09-25 |
| AZN | AstraZeneca | — | A Study to Assess the Effect of Ticagrelor in Reducing the Number of Days With Pain in Patients With Sickle Cell DiseaseNCT02482298results2017-12-14Change in Proportion of Days With Pain Due to Sickle Cell Disease as Measured by an eDiary | Phase 2 | Completed | Drug: Ticagrelor, Placebo | Sickle Cell Disease | 87 | 2015-07-09 | 2016-11-16actual | — | 2018-12-19 |
| AZN | AstraZeneca | — | A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are AdministeredNCT03126695Maximum observed plasma concentration (Cmax) | Phase 1 | Completed | Drug: Ticagrelor granule, Ticagrelor pediatric tablets, Ticagrelor pediatric tablets suspended in water, Ticagrelor immediate release (IR) tablets (Commercial tablet) | Sickle Cell Disease | 44 | 2017-05-12 | 2017-07-24actual | — | 2017-08-02 |
| LLY | Eli Lilly and Company | $1.15T | A Study of Prasugrel in Pediatric Participants With Sickle Cell DiseaseNCT01476696results2013-12-11Pharmacokinetics: Area Under the Concentration-Time Curve (AUC) of Prasugrel Active Metabolite (Pras-AM) | Phase 2 | Completed | Drug: Prasugrel | Sickle Cell Disease | 33 | Nov 2011 | Nov 2012actual | — | 2014-02-13 |
| LLY | Eli Lilly and Company | $1.15T | A Relative Bioavailability Study of a Prasugrel Orally Disintegrating TabletNCT01430091results2012-11-06Pharmacokinetics: Area Under the Concentration-Time Curve From Time Zero to the Last Measureable Concentration (AUC[0-tlast]) of Prasugrel's Active Metabolite (PRAS-AM) | Phase 1 | Completed | Drug: Prasugrel (clinical formulation), Prasugrel (Orally Disintegrating Tablet [ODT]) | Sickle Cell Disease | 18 | Sep 2011 | Oct 2011actual | — | 2012-11-06 |
| LLY | Eli Lilly and Company | $1.15T | Prasugrel Versus Placebo in Adult Sickle Cell DiseaseNCT01167023results2012-05-03Percentage of Participants With Hemorrhagic Events Requiring Medical Intervention During the Treatment Duration | Phase 2 | Completed | Drug: Prasugrel, Placebo | Sickle Cell Anemia | 62 | Jul 2010 | Jun 2011actual | — | 2012-05-03 |
| LLY | Eli Lilly and Company | $1.15T | An Assessment of Prasugrel on Healthy Adults and Sickle Cell AdultsNCT01178099results2012-02-15Area Under the Plasma Concentration-Time Curve (AUC) From Time of Dosing Through the Sampling Time of the Last Quantifiable Concentration [AUC(0-tlast)] for Prasugrel's Active Metabolite, R-138727 | Phase 1 | Completed | Drug: Prasugrel | Anemia, Sickle Cell | 26 | Jul 2010 | Jan 2011actual | — | 2012-02-15 |
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.
How Sickle Cell Disease is matched — 5 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.
- Sickle Cell Disease
- Sickle Cell Anemia
- Anemia, Sickle Cell
- Vaso-occlusive Pain Episode in Sickle Cell Disease
- Healthy Volunteers Sickle Cell Disease, Thalassemia
Permalink · All conditions · Catalyst calendar · Every registered study · Search the whole pipeline for “Sickle Cell Disease” (a text search, wider than this page)