Thalassemia Clinical Trials

61 recruitingLast updated: June 18, 2026

There are 61 actively recruiting thalassemia clinical trials across 39 countries. Studies span Phase 2, Not Applicable, Phase 1, Early Phase 1, Phase 3, Phase 4. Top locations include Nanning, Guangxi, China, New York, New York, United States, Tianjin, Tianjin Municipality, China. Updated daily from ClinicalTrials.gov.


Thalassemia Trials at a Glance

61 actively recruiting trials for thalassemia are listed on ClinicalTrialsFinder across 6 cities in 39 countries. The largest study group is Phase 2 with 16 trials, with the heaviest enrollment activity in Nanning, New York, and Tianjin. Lead sponsors running thalassemia studies include Institute of Hematology & Blood Diseases Hospital, China, Shenzhen Hemogen, and National Heart, Lung, and Blood Institute (NHLBI).

Browse thalassemia trials by phase

Treatments under study

About Thalassemia Clinical Trials

Looking for clinical trials for Thalassemia? There are currently 18 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Thalassemia trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Thalassemia clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 61 trials

Recruiting
Phase 1Phase 2

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Beta-ThalassemiaSickle Cell Disease
National Heart, Lung, and Blood Institute (NHLBI)90 enrolled1 locationNCT07599176
Recruiting

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

Sickle Cell DiseaseMalariaAlpha and Beta Thalassemia+1 more
National Institute of Allergy and Infectious Diseases (NIAID)300 enrolled1 locationNCT03937817
Recruiting
Phase 3

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Sickle Cell DiseaseThalassemia
Novo Nordisk A/S480 enrolled105 locationsNCT06609226
Recruiting
Phase 2

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Sickle Cell DiseaseThalassemiaThalassemia (beta)
Memorial Sloan Kettering Cancer Center24 enrolled6 locationsNCT05736419
Recruiting

A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East

Thalassemia
Bristol-Myers Squibb200 enrolled2 locationsNCT07215975
Recruiting
Phase 1

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

Sickle Cell DiseaseHb-SS DiseaseHemoglobin S+3 more
National Heart, Lung, and Blood Institute (NHLBI)25 enrolled1 locationNCT05904093
Recruiting
Phase 3

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-ThalassemiaMyelodysplastic Syndromes (MDS)Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
Celgene665 enrolled143 locationsNCT04064060
Recruiting
Phase 2

Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin

AlbuminuriaSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
University of Illinois at Chicago20 enrolled1 locationNCT07175051
Recruiting
Not Applicable

Autologous Testicular Tissue Transplantation

CancerHematologic DiseasesSickle Cell Thalassemia
Universitair Ziekenhuis Brussel5 enrolled1 locationNCT05414045
Recruiting
Phase 1Phase 2

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia
Children's Hospital of Philadelphia12 enrolled1 locationNCT06364774
Recruiting
Phase 3

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Beta-ThalassemiaSickle Cell DiseaseHematologic Diseases+4 more
Vertex Pharmaceuticals Incorporated26 enrolled6 locationsNCT05477563
Recruiting
Phase 2

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Regeneron Pharmaceuticals95 enrolled26 locationsNCT06421636
Recruiting
Phase 2

A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia

Thalassemia
Kind Pharmaceuticals LLC64 enrolled5 locationsNCT06302491
Recruiting

Atrial Fibrillation in Beta-Thalassemia

Atrial FibrillationThalassemia
University Hospital of Ferrara350 enrolled1 locationNCT05508932
Recruiting
Early Phase 1

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

Sickle Cell DiseaseThalassemiaHemoglobinopathy
National Heart, Lung, and Blood Institute (NHLBI)100 enrolled1 locationNCT06313398
Recruiting
Not Applicable

Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia

Thalassemia MajorThalassemia IntermediaGrowth Delay+1 more
Institute of Hematology & Blood Diseases Hospital, China369 enrolled1 locationNCT06931912
Recruiting
Phase 1

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Beta-ThalassemiaSickle Cell DiseaseSickle Cell Anemia (HbSS, or HbSβ-thalassemia0)+1 more
Daniel Bauer10 enrolled1 locationNCT06647979
Recruiting

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

ThrombophiliaThrombosisSickle Cell Disease+10 more
American Thrombosis and Hemostasis Network3,000 enrolled71 locationsNCT04398628
Recruiting
Phase 2

Evaluating the Clinical Efficacy and Safety of Luspatercept Combined With Thalidomide in the Treatment of β-TDT Patients

Transfusion-dependent β-thalassemia Patients
Rongrong Liu78 enrolled8 locationsNCT07338344
Recruiting
Phase 2

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Beta-Thalassemia
Celgene99 enrolled26 locationsNCT04143724