A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: * Participants receiving luspatercept on a parent protocol at the t...
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dep...
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologou...
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)
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers t...
Top Cities for Thalassemia Clinical Trials
Thalassemia clinical trials are recruiting across 27 cities. Here are the cities with the most active studies:
About Thalassemia
Thalassemia is an inherited blood disorder causing the body to produce abnormal or insufficient hemoglobin. Severe forms (thalassemia major) require regular blood transfusions. Treatment includes transfusions, iron chelation therapy, and potentially curative gene therapy.
Clinical trials are advancing new treatments for thalassemia. Currently, 4 studies are recruiting a combined 814 participants across the United States. Research is being conducted by 3 organizations including Celgene, Vertex Pharmaceuticals Incorporated, Memorial Sloan Kettering Cancer Center.
2026 Thalassemia Research Landscape
As of March 2026, the thalassemia clinical trial landscape includes 4 actively recruiting studies across 27 cities in the United States. These studies are collectively seeking 814 participants, with an average enrollment target of 204 per study.
Research is being led by 3 different organizations, including Celgene, Vertex Pharmaceuticals Incorporated, Memorial Sloan Kettering Cancer Center.
Geographically, thalassemia trials are most concentrated in New York, New York (3 trials); Los Angeles, California (2 trials); Guangzhou, Guangdong (2 trials); Nanning, GX (2 trials); Oakland, California (1 trial) and 7 other cities.
Featured Thalassemia Studies
Highlighted recruiting studies for thalassemia, selected by enrollment size and research scope.
A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants: * Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit from continuing tre...
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to \<18 years with two dose escalation cohorts, followed by a dose expa...
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.
Frequently Asked Questions About Thalassemia Clinical Trials
Are there thalassemia clinical trials near me?
Yes, there are 4 thalassemia clinical trials currently recruiting across 27+ cities in the United States, including New York, New York; Los Angeles, California; Guangzhou, Guangdong. Browse the studies above to find one at a location convenient for you.
How do I join a thalassemia clinical trial?
To join a thalassemia clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are thalassemia clinical trials free?
Yes, participation in thalassemia clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of thalassemia treatments are being studied?
Current thalassemia clinical trials are testing a range of approaches. These include new drugs, combination therapies, medical devices, and other interventions sponsored by 3 research organizations.
Is it safe to participate in thalassemia clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated March 2, 2026 from ClinicalTrials.gov
About This Data
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated March 2, 2026 · Data from ClinicalTrials.gov