117 completed, 37 terminated, 27 recruiting, 19 unknown. 230 Phase 3, 39 Phase 2.
Follow Anemia, Hemolytic: new trials by email Literature Review of Anemia, Hemolytic trials Research Report on Anemia, Hemolytic
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 |
|---|---|---|---|---|---|---|
| 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of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype Exa-cel - Sickle Cell Disease | Phase 3 | Withdrawn | Vertex Pharmaceuticals Incorporated | 2027-07-31 | 2026-03-25 | |
| A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients. FXS6837 Capsule - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Not yet recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 20 | 2027-01-26 | 2026-11-04 |
| A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH. FXS6837 Capsule, Eculizumab infusion - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Not yet recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 90 | 2026-12-10 | 2026-08-05 |
| A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy VSA012, Eculizumab (Soliris®) - Paroxysmal Nocturnal Hemoglobinuria, PNH | Phase 3 | Not yet recruiting | Bisirna Therapeutics (Suzhou) Co., Ltd. | 70 | 2026-10-29 | 2026-09-11 |
| Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia Thalidomide, Danazol, Prednisolone - Beta-Thalassemia, Thalassemia | Phase 3 | Recruiting | Khyber Medical University Peshawar | 172 | 2026-10-12 | 2026-10-21 |
| Evaluation of the Efficacy of Iloprost in the Management of Vaso-occlusive Crises in Adult Patients With Sickle Cell Disease Iloprost, Placebo - Vaso-Occlusive Crises, Sickle Cell Disease | Phase 3 | Not yet recruiting | University Hospital, Rouen | 144 | 2026-09-01 | 2026-03-27 |
| A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) Mitapivat Matched Placebo, Mitapivat - Sickle Cell Disease | Phase 3 | Recruiting | Agios Pharmaceuticals, Inc. | 159 | 2026-07-21 | 2026-10-28 |
| Lidocaine for Opioid Sparing in Vaso-occlusive Crisis of Sickle Cell Disease Lidocain, Placebo, Standard of care - Vaso-Occlusive Pain Episode in Sickle Cell Disease | Phase 3 | Recruiting | Nantes University Hospital | 104 | 2026-07-03 | 2026-09-09 |
| A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently Pozelimab, Cemdisiran - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Recruiting | Regeneron Pharmaceuticals | 35 | 2026-06-11 | 2026-08-05 |
| A Long-term Efficacy and Safety of NTQ5082 Capsules NTQ5082 capsules 200 mg - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Not yet recruiting | Nanjing Chia-tai Tianqing Pharmaceutical | 78 | 2026-06-01 | 2025-09-17 |
| Analgesic Effect of Parecoxib Versus Morphine in SCD Patients Presenting to the Emergency Department Parecoxib, Morphine - Sickle-Cell Disease with Crisis | Phase 3 | Not yet recruiting | Oman Medical Speciality Board | 226 | 2026-05-01 | 2025-03-20 |
| Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia Pharmacokinetic based dosage change, Pharmacokinetic dosing - Sickle Cell Disease (SCD) | Phase 2 / Phase 3 | Recruiting | Yves Pastore | 29 | 2026-02-03 | 2026-08-21 |
| Optimal Timing of Ketamine Initiation for SCD Pain Ketamine hydrochloride injection, Placebo - Sickle Cell Disease, Sickle Cell Crisis | Phase 3 | Not yet recruiting | Boston Children's Hospital | 90 | 2026-02-01 | 2026-03-09 |
| Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease PCV21, 20vPCV - Sickle Cell Disease | Phase 3 | Recruiting | Sanofi | 100 | 2026-01-20 | 2026-04-15 |
| A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients NTQ5082 capsules 200 mg, Eculizumab Injection - Paroxysmal Nocturnal Hemoglobinuria | Phase 3 | Not yet recruiting | Nanjing Chia-tai Tianqing Pharmaceutical | 78 | 2025-11-01 | 2025-09-17 |
| Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients LNP023 - Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | Recruiting | Novartis Pharmaceuticals | 12 | 2025-10-28 | 2026-08-07 |
| Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia Iron chelating intervention, N Acetyl cysteine 600mg, Alpha Lipoic Acid 600 MG Oral Tablets - Beta Thalassemia | Phase 3 | Not yet recruiting | Tanta University | 66 | 2025-08-30 | 2025-09-05 |
| Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Patients With Sickle Cell Disease Tocilizumab (RoActemra®, 20 mg/mL)., Placebo (NaCl 0.9%) - Sickle Cell Disease, Acute Chest Syndrome | Phase 3 | Recruiting | Assistance Publique - Hôpitaux de Paris | 130 | 2025-08-27 | 2026-06-24 |
| A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia rilzabrutinib, placebo - Autoimmune Haemolytic Anaemia | Phase 3 | Recruiting | Sanofi | 90 | 2025-08-18 | 2026-10-20 |
| The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease Rilzabrutinib, Placebo - Sickle Cell Disease | Phase 3 | Recruiting | Sanofi | 192 | 2025-08-12 | 2026-10-15 |
Showing 20 of 230. Sign in to see all of them.
Anemia, Sickle Cell (107) · Hemoglobinuria, Paroxysmal (50) · beta-Thalassemia (32) · Vaso-Occlusive Crises (26) · Hematologic Diseases (20) · Thalassemia (19) · Anemia (11) · Atypical Hemolytic Uremic Syndrome (10) · Hemoglobinopathies (10) · Iron Overload (9) · Anemia, Hemolytic, Autoimmune (8) · Hemolysis (8) · Acute Chest Syndrome (7) · Genetic Diseases, Inborn (6)
Hemic and Lymphatic Diseases (230) · Anemia (219) · Hematologic Diseases (210) · Anemia, Hemolytic, Congenital (154) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (154) · Genetic Diseases, Inborn (148) · Hemoglobinopathies (139) · Bone Marrow Diseases (54) · Myelodysplastic Syndromes (48) · Pathological Conditions, Signs and Symptoms (28) · Thalassemia (24) · Female Urogenital Diseases and Pregnancy Complications (18) · Female Urogenital Diseases (17) · Male Urogenital Diseases (17)
eculizumab (26) · Hydroxyurea (17) · ravulizumab (10) · Deferasirox (9) · exagamglogene autotemcel (7) · iptacopan (7) · Deferoxamine (6) · Deferiprone (5) · voxelotor (5) · Blood Transfusion (4) · Docosahexaenoic Acids (4) · Morphine (4) · Thalidomide (4) · mitapivat (4) · Alemtuzumab (3)
Novartis Pharmaceuticals (15) · Alexion Pharmaceuticals, Inc. (14) · Pfizer (11) · Assistance Publique - Hôpitaux de Paris (9) · Ain Shams University (7) · Vertex Pharmaceuticals Incorporated (7) · National Heart, Lung, and Blood Institute (NHLBI) (6) · Hoffmann-La Roche (5) · Regeneron Pharmaceuticals (5) · Agios Pharmaceuticals, Inc. (4) · Sanofi (4) · Actelion (3) · Children's Hospital Medical Center, Cincinnati (3) · Genetix Biotherapeutics Inc. (3) · Indiana University (3)
United States (102) · United Kingdom (60) · France (58) · Italy (57) · Canada (44) · Germany (42) · Brazil (37) · Spain (37) · Turkey (Türkiye) (34) · Netherlands (29) · Belgium (28) · China (26) · Egypt (25) · Japan (24) · South Korea (21)
Michael R. DeBaun, MD, MPH (3) · Russell E. Ware, MD, PhD (3) · Ahmed F Mahmoud Hussein, MS.c (2) · Anders Berggren, MD, PhD (2) · Charles Quinn, MD (2) · Claudia Morris, MD (2) · Donald Brambilla (2)
PMID 37975597, PMID 37254256, PMID 20175775, PMID 33783815, PMID 34219224, PMID 39535306, PMID 40277027, PMID 40694294, PMID 17634466, PMID 19721013, PMID 30952488, PMID 31974203, PMID 33283989, PMID 34175041, PMID 34526067, PMID 35981565, PMID 36594181, PMID 38477987, PMID 38727860, PMID 38775255, PMID 39079163, PMID 40447351, PMID 40720060, PMID 40857358, PMID 40862696, PMID 40978458, PMID 42252696, PMID 42274009, PMID 12966125, PMID 16382063, PMID 16990386, PMID 17634458, PMID 17634459, PMID 17702897, PMID 17951527, PMID 18055865, PMID 18055982, PMID 18071721, PMID 18343277, PMID 18351337, PMID 18478575, PMID 19222413, PMID 19471252, PMID 19621454, PMID 19813252, PMID 19856395, PMID 19880138, PMID 19951979, PMID 19996412, PMID 20117244
2023 (11), 2019 (8), 2017 (7), 2016 (4), 2004 (1), 2014 (1), 2025 (1), K23HL136787 (1), N01HB07150 (1), N01HB07151 (1), N01HB07152 (1), N01HB07153 (1), N01HB07154 (1), N01HB07155 (1), N01HB07156 (1)