Anemia, Hemolytic, Congenital: Clinical Trials

823 completed, 181 recruiting, 172 unknown, 146 terminated. 394 Phase 2, 352 Not applicable, 221 Phase 1, 154 Phase 3, 77 Phase 4.

By phase and status

Phase	Trials	Status
Phase 2	394	Completed 196, Terminated 54, Recruiting 47, Unknown 36, Active not recruiting 31, Withdrawn 23, Not yet recruiting 6, Enrolling by invitation 1
Not applicable	352	Completed 201, Recruiting 48, Unknown 40, Terminated 23, Not yet recruiting 18, Active not recruiting 13, Withdrawn 6, Suspended 3
Phase 1	221	Completed 114, Recruiting 28, Terminated 28, Active not recruiting 21, Unknown 12, Withdrawn 11, Not yet recruiting 6, Enrolling by invitation 1
Phase 3	154	Completed 72, Terminated 31, Unknown 18, Recruiting 15, Active not recruiting 7, Not yet recruiting 6, Withdrawn 4, Enrolling by invitation 1
Phase 4	77	Completed 40, Unknown 11, Terminated 7, Recruiting 6, Withdrawn 6, Active not recruiting 3, Not yet recruiting 3, Enrolling by invitation 1
Early Phase 1	23	Completed 8, Recruiting 4, Unknown 3, Not yet recruiting 2, Terminated 2, Withdrawn 2, Active not recruiting 1, Enrolling by invitation 1

Follow Anemia, Hemolytic, Congenital: new trials by email Literature Review of Anemia, Hemolytic, Congenital trials Research Report on Anemia, Hemolytic, Congenital

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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Safety and Efficacy of KL003 Cell Injection in Severe Sickle Cell Disease KL003 Cell Injection - Sickle Cell Disease	Not applicable	Suspended	Institute of Hematology & Blood Diseases Hospital, China	3	2028-03-01	2026-11-05
Self-administered Acupressure in Patients With Sickle Cell Disease Verum Acupressure (using AcuWand), Sham Acupressure (using AcuWand) - Sickle Cell Disease (SCD)	Not applicable	Not yet recruiting	University of Cincinnati	300	2027-10-01	2026-10-15
Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease Individuation and Perspective-Taking (IPT), pediatric SCD pain management education session - Sickle Cell Disease, Sickle Cell Anemia in Children	Not applicable	Not yet recruiting	Connecticut Children's Medical Center	30	2027-09-01	2026-04-29
Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype Exa-cel - Sickle Cell Disease	Phase 3	Withdrawn	Vertex Pharmaceuticals Incorporated		2027-07-31	2026-03-25
Data Collection For Patients With Sickle Cell Disease data collection - Data Collection, Sickle Cell Disease		Not yet recruiting	M.D. Anderson Cancer Center	500	2027-04-06	2026-11-03
MBI for Pain and Sleep in Adolescents and Young Adults With Sickle Cell Disease Mindfulness-based intervention - Chronic Pain, Sickle Cell Disease	Not applicable	Not yet recruiting	University of Illinois at Chicago	48	2027-02-01	2026-09-21
Focused Ultrasound Neuromodulation for Chronic Pain Focused PCM-Guided CMN LIFU, Unfocused Control LIFU - Chronic Pain, Sickle Cell Disease	Not applicable	Not yet recruiting	University of Minnesota	80	2027-01-04	2026-11-02
Heterologous Cord Blood-Derived Red Blood Cell for Transfusion in Extremely Preterm Infants Cord Blood Red Blood Cells, Standard Adult Donor Red Blood Cells - Extremely Premature Infant, Anemia Neonatal	Not applicable	Not yet recruiting	Hospital Clinic of Barcelona	176	2027-01-01	2026-03-31
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease Buprenorphine Buccal Film [Belbuca] - Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis	Phase 1	Not yet recruiting	Natasha Archer	25	2027-01-01	2026-11-04
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector Gene-modified autologous hematopoeitic stem cells - Beta-Thalassemia	Not applicable	Not yet recruiting	Shenzhen Geno-Immune Medical Institute	20	2026-12-31	2026-11-02
Omega-3-Fatty Acids in Children With Sickle Cell Disease Flaxseed - Sickle Cell Disease (SCD)	Not applicable	Not yet recruiting	University of Alabama at Birmingham	20	2026-12-01	2026-08-21
Minocycline In Neurocognitive Outcomes - Sickle Cell Disease Minocycline, Placebo - Sickle Cell Disease, Cognitive Impairment	Phase 1	Not yet recruiting	University of Cincinnati	30	2026-12-01	2026-11-03
Hypoxic Red Blood Cells in Sickle Cell Anemia Hemanext ONE System, Conventional RBCs - Sickle Cell Anaemia, Sickle Cell Anemia Crisis	Not applicable	Not yet recruiting	Hemanext	48	2026-11-30	2026-11-02
The Effect of Motivational Interviewing on Treatment Adherence and Self-Efficacy Among Omani Adults With Thalassemia Motivational Interviewing - Beta-Thalassemia Major, Transfusion-Dependent Thalassemia	Not applicable	Not yet recruiting	Oman Ministry of Health	45	2026-11-01	2026-06-22
Virtual Reality for SCD VOC Sham 2D Headset Control, Passive Immersive 3D Virtual Reality, Active Immersive Interactive 3D Virtual Reality - Sickle Cell Disease	Not applicable	Recruiting	University of Maryland, Baltimore	50	2026-11-01	2026-10-16
Feasibility and Acceptability of Amani Amani - Sickle Cell Disease	Phase 1 / Phase 2	Not yet recruiting	Massachusetts General Hospital	40	2026-11-01	2026-08-07
Inflammatory Markers and Pain in Sickle Cell Disease Sickle Cell Disease		Not yet recruiting	Assiut University	75	2026-11-01	2026-10-08
Transcranial Photobiomodulation Treatment in Patients With Sickle Cell Disease Active Transcranial Photobiomodulation (TPBM), Sham Transcranial photobiomodulation (TPBM) - Sickle Cell Disease	Not applicable	Not yet recruiting	St. Jude Children's Research Hospital	60	2026-11-01	2026-09-10
Efficacy and Safety of SIL-8301 for Control of Hemolysis in a Uniform Sickle Cell Disease Endotype Senicapoc, Placebo - Sickle Cell Disease, Sickle Cell Anaemia	Phase 2	Recruiting	Biossil Inc.	65	2026-11-01	2026-11-02
Native T1 Mapping and ECV in Chronic Hemolytic Anemia Hemolytic Anemia, Thalassemia		Not yet recruiting	Assiut University	54	2026-10-20	2026-10-20

Showing 20 of 1537. Sign in to see all of them.

Narrower conditions

Anemia, Sickle Cell (1120)  ·  beta-Thalassemia (264)  ·  Thalassemia (220)  ·  Vaso-Occlusive Crises (103)  ·  Hemoglobinopathies (70)  ·  Hematologic Diseases (69)  ·  Iron Overload (65)  ·  Pain (57)  ·  Anemia (55)  ·  Sickle Cell Trait (43)  ·  Glucosephosphate Dehydrogenase Deficiency (41)  ·  Acute Chest Syndrome (40)  ·  Genetic Diseases, Inborn (34)  ·  Anemia, Diamond-Blackfan (33)  ·  Hemoglobin SC Disease (32)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1536)  ·  Hemic and Lymphatic Diseases (1536)  ·  Anemia, Hemolytic (1514)  ·  Genetic Diseases, Inborn (1503)  ·  Anemia (1482)  ·  Hematologic Diseases (1468)  ·  Hemoglobinopathies (1414)  ·  Pathological Conditions, Signs and Symptoms (236)  ·  Thalassemia (229)  ·  Nutritional and Metabolic Diseases (185)  ·  Metabolic Diseases (171)  ·  Pathologic Processes (127)  ·  Signs and Symptoms (126)  ·  Respiratory Tract Diseases (113)

Interventions

Hydroxyurea (88)  ·  Deferasirox (39)  ·  Alemtuzumab (36)  ·  Cyclophosphamide (35)  ·  fludarabine (34)  ·  Sirolimus (29)  ·  fludarabine phosphate (27)  ·  Busulfan (25)  ·  Blood Specimen Collection (24)  ·  Deferoxamine (23)  ·  voxelotor (23)  ·  Whole-Body Irradiation (22)  ·  Deferiprone (21)  ·  Mycophenolic Acid (19)  ·  Tacrolimus (16)

Sponsors

National Heart, Lung, and Blood Institute (NHLBI) (64)  ·  Assistance Publique - Hôpitaux de Paris (52)  ·  St. Jude Children's Research Hospital (45)  ·  Novartis Pharmaceuticals (43)  ·  Children's Hospital Medical Center, Cincinnati (31)  ·  Duke University (28)  ·  Emory University (28)  ·  Pfizer (27)  ·  Assiut University (25)  ·  HaEmek Medical Center, Israel (19)  ·  UCSF Benioff Children's Hospital Oakland (17)  ·  Ain Shams University (15)  ·  University of Illinois at Chicago (14)  ·  Washington University School of Medicine (14)  ·  Vanderbilt University Medical Center (13)

Countries

United States (830)  ·  France (148)  ·  United Kingdom (98)  ·  Italy (95)  ·  Canada (78)  ·  Egypt (76)  ·  China (68)  ·  Turkey (Türkiye) (60)  ·  Lebanon (58)  ·  Greece (45)  ·  Brazil (40)  ·  Israel (37)  ·  Thailand (37)  ·  Belgium (36)  ·  Nigeria (36)

Top investigators

John F Tisdale, M.D. (16)  ·  Ariel Koren, MD (12)  ·  Swee Lay Thein, M.D. (12)  ·  Lakshmanan Krishnamurti, MD (9)  ·  Janet Kwiatkowski, MD (7)  ·  Russell Ware, MD, PhD (7)  ·  Carina Levin, MD (6)  ·  Elliott Vichinsky, MD (6)  ·  Michael R DeBaun, MD, MPH (6)

Results publications

PMID 37975597, PMID 36240296, PMID 37254256, PMID 38775255, PMID 40569673, PMID 41026975, PMID 42133908, PMID 26589907, PMID 34175041, PMID 20175775, PMID 24916507, PMID 33661274, PMID 36860093, PMID 36975201, PMID 38866240, PMID 39382077, PMID 39428602, PMID 39659429, PMID 41809202, PMID 42705233, PMID 17634466, PMID 19721013, PMID 23416852, PMID 29931453, PMID 30617198, PMID 30655275, PMID 30952488, PMID 31799940, PMID 32384147, PMID 33283989, PMID 33570654, PMID 34000072, PMID 34922648, PMID 35019238, PMID 35075288, PMID 35120836, PMID 35981565, PMID 36123234, PMID 36355805, PMID 36529836, PMID 36637054, PMID 36890729, PMID 37704579, PMID 38249565, PMID 38361924, PMID 38538687, PMID 39615812, PMID 40010689, PMID 40670924, PMID 40800810

NIH funding

2019 (10), U01HL065238 (8), U54HL070587 (8), 2023 (7), 2014 (6), 2017 (6), 2018 (6), U01HL133996 (4), 2025 (3), 2004 (2), 2016 (2), K23HL094376 (2), K23HL148310 (2), K23HL150232 (2), K24AT009893 (2)