Disease Landscape

Thalassemia Clinical Trials

Data updated daily from ClinicalTrials.gov — last refresh 2026-08-16
120
Active Trials
60
Recruiting
10
Phase 3
70
Sponsors
27
Changes, 30 Days
What Changed in the Last 30 Days
DetectedTrialChange
2026-08-14 NCT03653338
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD)… · Paul Szabolcs
date change Primary completion pushed: 2026-08-01 -> 2028-08-01
2026-08-14 NCT03653338
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD)… · Paul Szabolcs
date change Completion pushed: 2027-08-01 -> 2028-08-01
2026-08-13 NCT05777993
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an… · Agios Pharmaceuticals, Inc.
Primary endpoint(s) modified
2026-08-13 NCT05777993
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an… · Agios Pharmaceuticals, Inc.
Enrollment increased: 6 -> 59 participants
2026-08-13 NCT05777993
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an… · Agios Pharmaceuticals, Inc.
Trial sites expanded: 4 -> 23 locations
2026-08-12 NCT06743113
Hypoxic Red Blood Cells in Sickle Cell Anemia · Hemanext
date change Primary completion pushed: 2028-04 -> 2029-04
2026-08-12 NCT06743113
Hypoxic Red Blood Cells in Sickle Cell Anemia · Hemanext
date change Completion pushed: 2028-09 -> 2029-10
2026-08-09 NCT04310059
Effect of Folic Acid Supplementation in Pregnant Women Having Thalassaemia Trait · The University of Hong Kong
overall status: Not Yet Recruiting → Unknown
2026-08-07 NCT07673302
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia · Riphah International University
Recruitment opened
2026-08-04 NCT07517133
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in… · Agios Pharmaceuticals, Inc.
Recruitment opened
2026-08-04 NCT07506863
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in… · Agios Pharmaceuticals, Inc.
Recruitment opened
2026-08-01 NCT04691323
The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit… · Ann & Robert H Lurie Children's Hospital of Chicago
Trial completed
2026-08-01 NCT04691323
The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit… · Ann & Robert H Lurie Children's Hospital of Chicago
date change Primary completion moved earlier: 2025-06-01 -> 2024-06-18
2026-08-01 NCT04691323
The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit… · Ann & Robert H Lurie Children's Hospital of Chicago
date change Completion moved earlier: 2025-12-01 -> 2025-05-29
2026-08-01 NCT04688411
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell… · Ann & Robert H Lurie Children's Hospital of Chicago
Trial completed
2026-08-01 NCT04688411
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell… · Ann & Robert H Lurie Children's Hospital of Chicago
Enrollment reduced: 40 -> 36 participants
2026-08-01 NCT04688411
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell… · Ann & Robert H Lurie Children's Hospital of Chicago
date change Primary completion pushed: 2025-02-01 -> 2025-09-16
2026-08-01 NCT04688411
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell… · Ann & Robert H Lurie Children's Hospital of Chicago
date change Completion pushed: 2025-02-01 -> 2025-09-16
2026-07-29 NCT07003269
Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT · Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
overall status: Enrolling by Invitation → Completed
2026-07-29 NCT07003256
Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT · Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
overall status: Enrolling by Invitation → Completed
Showing 20 of 27 changes detected in the last 30 days.
New Sponsor Entrants, Last 90 Days
First PostedTrialSponsorPhase
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
2026-05-29 NCT07614217 Oral Health Status in Children With β-Thalassemia Major Cairo University NEW
Active Trials by Phase
Phase 4
3
Phase 3
10
Phase 2
19
Phase 1/2
11
Phase 1
9
Early Phase 1
9
Not specified
59
Active Trials — top 25 of 120
NCT IDTitleSponsorStatus
Phase 4
NCT07177300 Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations… Children's Hospital Medical Center, Cincinnati Recruiting
NCT02386800 CINC424A2X01B Rollover Protocol Novartis Pharmaceuticals Active, Not Recruiting
NCT05777993 A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an… Agios Pharmaceuticals, Inc. Enrolling by Invitation
Phase 3
NCT05477563 Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With… Vertex Pharmaceuticals Incorporated Recruiting
NCT04064060 A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other… Celgene Recruiting
NCT07506863 A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in… Agios Pharmaceuticals, Inc. Recruiting
NCT07517133 A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in… Agios Pharmaceuticals, Inc. Recruiting
NCT06609226 A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle… Novo Nordisk A/S Recruiting
NCT07157722 Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta… Tanta University Not Yet Recruiting
NCT05356195 Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With… Vertex Pharmaceuticals Incorporated Active, Not Recruiting
NCT04770753 A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With… Agios Pharmaceuticals, Inc. Active, Not Recruiting
NCT04770779 A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With… Agios Pharmaceuticals, Inc. Active, Not Recruiting
NCT04208529 A Long-term Follow-up Study in Participants Who Received CTX001 Vertex Pharmaceuticals Incorporated Enrolling by Invitation
Phase 2
NCT06657391 Evaluation of Recombinant Humanized Anti-CD25 Monoclonal Antibody for Preventing… Rongrong Liu Recruiting
NCT06490627 Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta… National Institute of Blood and Marrow Transplant (NIBMT), Pakistan Recruiting
NCT04099966 AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell… Mitchell Cairo Recruiting
NCT05693909 A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk… Pharmacosmos A/S Recruiting
NCT07338344 Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in… Rongrong Liu Recruiting
NCT06302491 A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia Kind Pharmaceuticals LLC Recruiting
NCT07175051 Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2… University of Illinois at Chicago Recruiting
NCT05736419 A Study of Immune Suppression Treatment for People With Sickle Cell Disease or… Memorial Sloan Kettering Cancer Center Recruiting
NCT04143724 Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta… Celgene Recruiting
NCT07680803 Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia Fondazione Telethon Recruiting
NCT02105766 Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation… National Heart, Lung, and Blood Institute (NHLBI) Active, Not Recruiting
NCT06490601 Long Term Beta Thalassemia Treatment: Findings From The Extension Period National Institute of Blood and Marrow Transplant (NIBMT), Pakistan Active, Not Recruiting
See all 120 active Thalassemia trials with filters →
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