TheraRadar
Landscape / Rare Disease
Data updated: Jun 28, 2026

Sickle Cell Disease Clinical Trial Landscape

Sickle Cell Disease is being studied across 366 clinical trials registered since 2008, with 109 programs currently active. The competitive pipeline includes 23 active Phase 3 trials, 61 active Phase 2 trials, and 17 active Phase 1 trials.

Top industry sponsors include Novartis, Forma Therapeutics, Inc., Vertex Pharmaceuticals Incorporated.

Trial activity

109 active / 366 total since 2008
Active by phase 23 Ph3 / 71 61 Ph2 / 189 17 Ph1 / 77 8 Ph4 / 29

Sponsor activity

Who is running trials now — green active, blue completed, red failed/terminated.

Sorted by active Active Done Failed
Novartis 5 5 1
Forma Therapeutics, Inc. 3 2 0
Vertex Pharmaceuticals Incorporated 3 1 1
Agios Pharmaceuticals, Inc. 3 0 1
Novo Nordisk 2 6 0
CSL Behring 2 1 0
Sanofi 2 0 0
Pfizer 1 8 11
Fulcrum Therapeutics 1 2 1
Theravia 1 3 0
Genetix Biotherapeutics Inc. 1 2 0
Disc Medicine, Inc 1 0 0
Kamau Therapeutics 1 0 0
Beam Therapeutics Inc. 1 0 0
Biossil Inc. 1 0 0

All 15 active Sickle Cell Disease sponsors

Unlock the remaining 7 sponsors with active / completed / failed counts — sortable and exportable.

Unlock with Pro

How the field has grown

New-trial starts peaked in 2022 (29 registered); 2025 saw 22. The right-hand chart shows median Phase 3 enrollment by start year — the number in parentheses is that year's Phase 3 trial count (53 in total), so single-trial years (and years with no Phase 3 starts) are obvious. Both are by trial start date; the current year is partial.

New trials started by year

2016
15
2017
24
2018
23
2019
20
2020
22
2021
25
2022
29
2023
18
2024
18
2025
22
2026
23

TheraRadar.com

Median Phase 3 enrollment by start year

2016 (3)
440
2017 (3)
120
2018 (4)
121
2019 (6)
209
2020 (4)
205
2021 (7)
270
2022 (10)
40
2023 (3)
120
2024 (1)
315
2025 (7)
130
2026 (5)
104

TheraRadar.com

Full trial pipeline

Every active and completed trial across Phase 1–4, with enrollment analytics. Sortable, filterable, exportable with Pro.

NCT06439082 RECRUITING
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
Novartis Pharmaceuticals n=315
NCT06975865 RECRUITING
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
Sanofi n=192
NCT06612268 RECRUITING
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
Novo Nordisk A/S n=408
NCT05031780 ACTIVE NOT RECRUITING
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Agios Pharmaceuticals, Inc. n=286
NCT03814746 ACTIVE NOT RECRUITING
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
Novartis Pharmaceuticals n=255
NCT07247188 RECRUITING
Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease
Sanofi n=100
NCT04624659 ACTIVE NOT RECRUITING
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
Forma Therapeutics, Inc. n=450
NCT07498309 NOT YET RECRUITING
Evaluation of the Efficacy of Iloprost in the Management of Vaso-occlusive Crises in Adult Patients With Sickle Cell Disease
University Hospital, Rouen n=144
NCT04208529 ENROLLING BY INVITATION
A Long-term Follow-up Study in Participants Who Received CTX001
Vertex Pharmaceuticals Incorporated n=160
NCT06609226 RECRUITING
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Novo Nordisk A/S n=480
NCT05477563 RECRUITING
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Vertex Pharmaceuticals Incorporated n=26
NCT05431088 RECRUITING
A Phase 2/3 Study in Adult and Adolescent Participants With SCD
Pfizer n=389
NCT05329649 ACTIVE NOT RECRUITING
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
Vertex Pharmaceuticals Incorporated n=13
NCT07450430 NOT YET RECRUITING
Optimal Timing of Ketamine Initiation for SCD Pain
Boston Children's Hospital n=90
NCT04084080 ACTIVE NOT RECRUITING
Sickle Cell Disease and CardiovAscular Risk - Red Cell Exchange Trial (SCD-CARRE)
University of Pittsburgh n=173
NCT07274254 NOT YET RECRUITING
Lidocaine for Opioid Sparing in Vaso-occlusive Crisis of Sickle Cell Disease
Nantes University Hospital n=104
NCT06989567 NOT YET RECRUITING
A Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis
Asklepion Pharmaceuticals, LLC n=120
NCT05285917 RECRUITING
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
Brown University n=400
NCT06818266 RECRUITING
Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Patients With Sickle Cell Disease
Assistance Publique - Hôpitaux de Paris n=130
NCT06761560 NOT YET RECRUITING
Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia
Yves Pastore n=29
NCT04293185 ACTIVE NOT RECRUITING
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
Genetix Biotherapeutics Inc. n=35
NCT05392894 RECRUITING
RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease
King's College Hospital NHS Trust n=120
NCT07656415 NOT YET RECRUITING
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Agios Pharmaceuticals, Inc. n=159
NCT05951205 WITHDRAWN
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype
Vertex Pharmaceuticals Incorporated
NCT05632354 TERMINATED
GBT021601-022: A Study of GBT021601 in Participants With Sickle Cell Disease (SCD)
Pfizer n=47
NCT04218084 TERMINATED
Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease
Pfizer n=236
NCT06561061 COMPLETED
Zinc for Infection Prevention in Sickle Cell Anemia-2 (ZIPS-2)
Indiana University n=100
NCT05470998 COMPLETED
L-Arginine in Children Having Sickle Cell Disease With Increased Tricuspid Regurgitant Jet Velocity
Tanta University n=50
NCT05348915 TERMINATED
A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease
Pfizer n=242
NCT04935879 COMPLETED
A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises
Pfizer n=241
NCT02813850 COMPLETED
Oxygen Therapy and Pregnancy in Sickle Cell Disease
Assistance Publique - Hôpitaux de Paris n=178
NCT03573882 TERMINATED
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Pfizer n=179
NCT04188509 TERMINATED
Open-Label Extension of Voxelotor
Pfizer n=162
NCT05084521 COMPLETED
Interest of Famotidine in Children With Sickle Cell Disease
Assistance Publique - Hôpitaux de Paris n=30
NCT03745287 COMPLETED
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Vertex Pharmaceuticals Incorporated n=63
NCT04839354 COMPLETED
Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial
Claudia R. Morris n=271
NCT05561140 TERMINATED
Resolution of Sickle Cell Leg Ulcers With Voxelotor
Pfizer n=88
NCT03789591 COMPLETED
Hydroxyurea Optimization Through Precision Study
Lifespan n=104
NCT04750707 COMPLETED
Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda ( BRAINSAFE-II )
Global Health Uganda LTD n=270
NCT05033314 TERMINATED
THromboprophylaxis In Sickle Cell Disease With Central Venous Catheters (THIS)
University Health Network, Toronto n=4
NCT05253781 COMPLETED
Low Dose Aspirin for Preventing Intrauterine Growth Restriction and Preeclampsia in Sickle Cell Pregnancy (PIPSICKLE)
University of Lagos, Nigeria n=476
NCT04927247 TERMINATED
A Study of a Single Dose of Inclacumab to Reduce Re-admission in Participants With Sickle Cell Disease and Recurrent Vaso-occlusive Crises
Pfizer n=72
NCT03704922 COMPLETED
Age of Blood in Sickle Cell Transfusion
University of North Carolina, Chapel Hill n=26
NCT04150757 TERMINATED
Intranasal Ketamine for Pain Control in Patients with Sickle Cell Disease and Vaso-occlusive Episode (VOE) in the PED
Abigail Nixon n=5
NCT05228834 TERMINATED
Voxelotor Neurocognitive Function Study
Pfizer n=1
NCT04844099 COMPLETED
Dihydroartemisinin-Piperaquine or Sulphadoxine-Pyrimethamine for the Chemoprevention of Malaria in Sickle Cell Anaemia
Liverpool School of Tropical Medicine n=723
NCT03036813 COMPLETED
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
Pfizer n=449
NCT03933397 TERMINATED
Comparing Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease
Duke University n=328
NCT00560261 COMPLETED
Evaluation of the Lung Capillary Blood Volume in Children With Sickle Cell Disease
Assistance Publique - Hôpitaux de Paris n=120
NCT01877837 COMPLETED
Stem Cell Transplantation for Sickle Cell Anemia
Hackensack Meridian Health n=30
NCT02675790 COMPLETED
Moderate Dose Hydroxyurea for Secondary Stroke Prevention in Children With Sickle Cell Disease in Sub-Saharan Africa
Vanderbilt University Medical Center n=120
NCT03615924 TERMINATED
Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease
AstraZeneca n=193
NCT02433158 TERMINATED
Safety of Rivipansel (GMI-1070) in the Treatment of One or More Vaso-Occlusive Crises in Hospitalized Subjects With Sickle Cell Disease
GlycoMimetics Incorporated n=154
NCT04301336 COMPLETED
Different Treatment Modalities in the Management of the Painful Crisis in Pediatric Sickle- Cell Anemia
Beni-Suef University n=350
NCT01179217 COMPLETED
A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
Emmaus Medical, Inc. n=230
NCT02560935 COMPLETED
Primary Prevention of Stroke in Children With SCD in Sub-Saharan Africa II
Vanderbilt University Medical Center n=440
NCT01425307 TERMINATED
Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea
Children's Hospital Medical Center, Cincinnati n=159
NCT04293172 WITHDRAWN
Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease.
AstraZeneca
NCT03417947 COMPLETED
Vitamin D Supplementation in Children With Sickle Cell Disease
St. Justine's Hospital n=42
NCT02179177 TERMINATED
Apixaban in Patients With Sickle Cell Disease
Nirmish Shah n=16
NCT03128515 COMPLETED
Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas
Indiana University n=187
NCT01794000 TERMINATED
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
Eli Lilly and Company n=341
NCT01976416 COMPLETED
Novel Use Of Hydroxyurea in an African Region With Malaria
Indiana University n=208
NCT01380197 COMPLETED
Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally
Children's Healthcare of Atlanta n=40
NCT01737814 COMPLETED
Evaluation of Purified Poloxamer 188 in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)
Mast Therapeutics, Inc. n=388
NCT02449616 COMPLETED
Evaluation of Repeat Administration of Purified Poloxamer 188
Mast Therapeutics, Inc. n=16
NCT01849016 COMPLETED
N-Acetylcysteine in Patients With Sickle Cell Disease
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) n=96
NCT01531387 TERMINATED
Sparing Conversion to Abnormal TCD (Transcranial Doppler) Elevation (SCATE)
Children's Hospital Medical Center, Cincinnati n=38
NCT01197417 COMPLETED
Intravenous Magnesium for Sickle Cell Vasoocclusive Crisis
Medical College of Wisconsin n=208
NCT00748423 COMPLETED
Effect of Inhaled Nitric Oxide in Acute Chest Syndrome (INOSTA Study)
Assistance Publique - Hôpitaux de Paris n=100
NCT00999245 TERMINATED
THE IMPROVE TRIAL: Improving Pain Management and Outcomes With Various Strategies of Patient-Controlled Analgesia (PCA)
Carelon Research n=38

Full Sickle Cell Disease Pipeline

Every trial across Phase 1–4, plus enrollment analytics. Sortable, filterable, exportable.

Unlock with Pro

Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only