Disease landscape

Thalassemia clinical trials

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123 active trials 60 recruiting 10 Phase 3 73 sponsors 13 changes in 30 days

Data updated daily from ClinicalTrials.gov, last refresh 2026-09-05

What changed in the last 30 days

2026-08-21
Primary completion pushed: 2027-09-16 → 2032-04-05
NCT02386800 CINC424A2X01B Rollover Protocol · Novartis Pharmaceuticals · Phase 4
2026-08-12
Primary completion pushed: 2028-04 → 2029-04
Completion pushed: 2028-09 → 2029-10
NCT06743113 Hypoxic Red Blood Cells in Sickle Cell Anemia · Hemanext
2026-08-14
Primary completion pushed: 2026-08-01 → 2028-08-01
Completion pushed: 2027-08-01 → 2028-08-01
NCT03653338 T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD)… · Paul Szabolcs · Phase 1/2
2026-08-13
Primary endpoint(s) modified
Enrollment increased: 6 → 59 participants
Trial sites expanded: 4 → 23 locations
NCT05777993 A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an… · Agios Pharmaceuticals, Inc. · Phase 4
2026-08-07
Recruitment opened
NCT07673302 Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia · Riphah International University
2026-08-28
Trial sites expanded: 1 → 4 locations
NCT07517133 A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in… · Agios Pharmaceuticals, Inc. · Phase 3
2026-08-20
Trial sites expanded: 2 → 13 locations
NCT07215975 A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent… · Bristol-Myers Squibb
2026-08-18
Trial sites expanded: 71 → 76 locations
NCT04398628 ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders · American Thrombosis and Hemostasis Network
2026-08-09
Trial status changed: Not Yet Recruiting → Unknown
NCT04310059 Effect of Folic Acid Supplementation in Pregnant Women Having Thalassaemia Trait · The University of Hong Kong

New sponsor entrants, last 90 days sponsors with their first trial in this area

First postedTrialSponsorPhase
2026-09-02 NCT07799571 Buprenorphine for the Management of Pain in Children With Sickle Cell Disease Natasha Archer new Phase 1
2026-08-31 NCT07796022 Clinical, Economic,and Psychosocial Burden of Thalassemia in Sohag Randa Elham Hamam new
2026-08-24 NCT07782931 Evaluate the Efficacy and Safety of Irochel for the Management of Iron Overload in… Hamdard University new Phase 3
2026-07-23 NCT07721480 Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia Prince of Wales Hospital, Shatin, Hong Kong new Phase 2
2026-07-13 NCT07698080 Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of… Hematology department of the 920th hospital new Phase 2
2026-07-02 NCT07680803 Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia Fondazione Telethon new Phase 2
2026-06-29 NCT07673302 Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia Riphah International University new
2026-06-22 NCT07660224 The Effect of Motivational Interviewing on Treatment Adherence and Self-Efficacy Among… Oman Ministry of Health new

Active trials by phase

Early Phase 1
9
Phase 1
10
Phase 1/2
11
Phase 2
19
Phase 3
10
Phase 4
3
Not charted: 61 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

Active trials top 25 of 123

NCT IDTitleSponsorStatusUpdated
Phase 4
NCT07177300 Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations… Children's Hospital Medical Center, Cincinnati Recruiting 2025-09-16
NCT02386800 CINC424A2X01B Rollover Protocol Novartis Pharmaceuticals Active, Not Recruiting 2026-08-20
NCT05777993 A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an… Agios Pharmaceuticals, Inc. Enrolling by Invitation 2026-08-12
Phase 3
NCT07506863 A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in… Agios Pharmaceuticals, Inc. Recruiting 2026-08-27
NCT07517133 A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in… Agios Pharmaceuticals, Inc. Recruiting 2026-08-27
NCT06609226 A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle… Novo Nordisk A/S Recruiting 2026-08-13
NCT04064060 A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other… Celgene Recruiting 2026-07-01
NCT05477563 Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With… Vertex Pharmaceuticals Incorporated Recruiting 2026-03-23
NCT04770753 A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With… Agios Pharmaceuticals, Inc. Active, Not Recruiting 2026-08-27
NCT04770779 A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With… Agios Pharmaceuticals, Inc. Active, Not Recruiting 2026-08-27
NCT04208529 A Long-term Follow-up Study in Participants Who Received CTX001 Vertex Pharmaceuticals Incorporated Enrolling by Invitation 2026-08-11
NCT05356195 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With… Vertex Pharmaceuticals Incorporated Active, Not Recruiting 2026-07-01
NCT07157722 Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta… Tanta University Not Yet Recruiting 2025-09-05
Phase 2
NCT07680803 Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia Fondazione Telethon Recruiting 2026-07-27
NCT04143724 Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta… Celgene Recruiting 2026-07-10
NCT05736419 A Study of Immune Suppression Treatment for People With Sickle Cell Disease or… Memorial Sloan Kettering Cancer Center Recruiting 2026-06-03
NCT07175051 Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2… University of Illinois at Chicago Recruiting 2026-05-19
NCT06302491 A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia Kind Pharmaceuticals LLC Recruiting 2026-02-27
NCT07338344 Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in… Rongrong Liu Recruiting 2026-01-13
NCT05693909 A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk… Pharmacosmos A/S Recruiting 2025-09-24
NCT04099966 AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell… Mitchell Cairo Recruiting 2025-08-08
NCT06490627 Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta… National Institute of Blood and Marrow Transplant (NIBMT), Pakistan Recruiting 2025-05-30
NCT06657391 Evaluation of Recombinant Humanized Anti-CD25 Monoclonal Antibody for Preventing… Rongrong Liu Recruiting 2024-10-28
NCT02105766 Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation… National Heart, Lung, and Blood Institute (NHLBI) Active, Not Recruiting 2026-08-18
NCT07721480 Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia Prince of Wales Hospital, Shatin, Hong Kong Not Yet Recruiting 2026-07-23
See all 123 active Thalassemia trials with filters →

Top sponsors by active trials in this area

Assiut University (7) · Agios Pharmaceuticals, Inc. (6) · National Heart, Lung, and Blood Institute (NHLBI) (6) · Bristol-Myers Squibb (4) · CorrectSequence Therapeutics Co., Ltd (4) · Institute of Hematology & Blood Diseases Hospital, China (4) · Shenzhen Hemogen (4) · Bioray Laboratories (3) · First Affiliated Hospital of Guangxi Medical University (3) · Hospital Universitari Vall d'Hebron Research Institute (3)

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