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...
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoi...
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...
Frequently Asked Questions
What clinical trials are available for Thalassemia?
There are currently 4 actively recruiting clinical trials for thalassemia. These studies are testing new treatments, therapies, and interventions at research sites across 22 cities.
How do I join a Thalassemia clinical trial?
To join a thalassemia clinical trial: 1) Browse the available studies above, 2) Click on a study that interests you, 3) Complete the eligibility form, and 4) A study coordinator will contact you.
Are clinical trials free for patients?
Yes, clinical trial participation is free. In most studies, the treatment, medical tests, and doctor visits related to the trial are provided at no cost. Some studies may also compensate for time and travel.
Can I leave a clinical trial if I change my mind?
Yes, participation in any clinical trial is completely voluntary. You can withdraw at any time, for any reason, without affecting your regular medical care.