Clinical Trials of Thalassemia Which Are Currently Recruiting U.S. Patients
(Last Updated: May 5, 2016)
The Cooley’s Anemia Foundation provides links to clinical trials for informational purposes only. The Foundation is not endorsing any one trial over others.
The clinical studies listed here are currently enrolling new thalassemia patients in the United States. The links below provide information from the clinicaltrials.gov website about the requirements for participating in these studies (“Eligibility”). They also provide information about where the trials are taking place (“Locations”). If you have questions about any of the trials listed below, please contact the CAF Patient Outreach Director, Sandy Gilbert, at 212-279-8090 (extension 207) for more information. You are also encouraged to speak to your doctor about any clinical trials which might be of interest to you.
The Cooley’s Anemia Foundation (CAF) provides these links for informational purposes only, and is not endorsing any one trial over the others. Please note that there are additional studies taking place in other countries which are not listed here. For example, Acceleron is currently conducting a study of luspatercept – which increases the production of mature red blood cells – in Europe. CAF will keep you updated on any important findings which emerge from these studies, and will let you know if any similar trials open up in the U.S.
I. Clinical Trials of Gene Therapy in Beta-Thalassemia Major
- A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects
- ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene (This trial is currently suspended in order to secure funding to treat the next cohort of participants. Patients with questions concerning this trial should contact Dr. Farid Boulad at email@example.com.)
II. Clinical Trials of Luspatercept
- An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia (BELIEVE)
III. Hepcidin Studies
IV. Clinical Trials of Iron Chelation Therapy
- Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
- Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload
V. Clinical Trials of Bone Marrow, Stem Cell or Cord Blood Transplantation
Please note that, while clinical trials are available to assess experimental approaches to transplantation, bone marrow transplantation is an approved procedure that is already widely available. If you are considering the possibility of a curative bone marrow transplant and you would like more information about this procedure, we recommend that you speak to one of the following experts:
- Dr. Alexis Thompson: 312-227-4816
- Dr. Mark Walters: 510-428-3374
- Dr. John Tisdale: 301-402-6497
VI. Clinical Studies of New Methods to Monitor Iron Levels
VII. Observational Studies of Patients with Thalassemia
VIII. Preclinical (Early-Stage) Research Studies in Thalassemia