Thalassemia Clinical Trials Currently Available to U.S. Patients

Thalassemia Clinical Trials Which Are Currently Recruiting U.S. Patients
(Last Updated: November 10, 2021)

The Cooley’s Anemia Foundation provides links to clinical trials for informational purposes only. The Foundation is not endorsing any one trial over others.

ClinicalTrialHeader2The clinical studies listed here are currently enrolling new thalassemia patients in the United States. The links below provide information from the 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.

ClinicalTrialHeader3The 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.

A. Interventional Studies

I. Clinical Trials of Gene Therapy in Beta-Thalassemia Major

II. Clinical Trials of Gene Editing in Transfusion-Dependent Beta Thalassemia

III. Clinical Trials for Patients with Non-Transfusion-Dependent Thalassemia

IV. Clinical Trials of Luspatercept

V. Clinical Trials of Hepcidin

VI. Clinical Trials of PDE9 Inhibition

VII. Clinical Trials of Fetal Hemoglobin Induction

VIII. Clinical Trials of Iron Chelation Therapy

IX. Clinical Trials in Alpha Thalassemia Major

X. Clinical Trials of Mitapivat and Etavopivat

XI. 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

B. Observational Studies

XII. Research Studies in Thalassemia

XIII. Preclinical (Early-Stage) Research Studies in Thalassemia

XIIV. Thalassemia-Related Trials in Healthy Volunteers

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