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.

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.

A. Interventional Studies

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

• 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 bouladf@mskcc.org.)

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

• A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
• A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent Beta-Thalassemia
• A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) – Full Text View – ClinicalTrials.gov

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

• A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants with Non-Transfusion-Dependent Thalassemia
• The BENeFiTS Trial in Beta Thalassemia Intermedia (PB04-001)
• A Study of IMR-687 in Subjects with Beta Thalassemia
• Fetal Hemoglobin Induction Treatment Metformin (FITMet) in non-transfusion dependent thalassemia

IV. Clinical Trials of Luspatercept

• Study of Safety and PK of Luspatercept (ACE-536) in Pediatric Subjects Who Require Regular RBC Transfusions Due to (β)-Thalassemia

V. Clinical Trials of Hepcidin

• Safety Study for Beta Thalassemia Subjects on PTG-300 (TRANSCEND)

VI. Clinical Trials of PDE9 Inhibition

• A Study of IMR-687 in Subjects with Beta Thalassemia

VII. Clinical Trials of Fetal Hemoglobin Induction

• Fetal Hemoglobin Induction Treatment Metformin (FITMet) in non-transfusion dependent thalassemia

VIII. Clinical Trials of Iron Chelation Therapy

• Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation

IX. Clinical Trials in Alpha Thalassemia Major

• In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

X. Clinical Trials of Mitapivat and Etavopivat

• A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) – Full Text View – ClinicalTrials.gov
• A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) – Full Text View – ClinicalTrials.gov
• A Study of FT-4202 in Patients With Thalassemia or Sickle Cell Disease

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

• Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia

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

• Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or Thalassemia

XIIV. Thalassemia-Related Trials in Healthy Volunteers

• Screening for Alpha Thalassemia in Healthy Volunteers with African Ancestry