Thalassemia Clinical Trials

Monday, January 13th, 2014 at 7:29 pm

Clinical Trials of Thalassemia Which Are Currently Recruiting U.S. Patients
(Last Updated: January 07, 2015)

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

II. Clinical Trials of Iron Chelation Therapy

• Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

• Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia

• Phase II Study to Investigate the Benefits of an Improved Deferasirox (Exjade) Formulation (Film-coated Tablet)

III. 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

IV. Clinical Studies of New Methods to Monitor Iron Levels

• High-Tc Susceptometer to Monitor Transfusional Iron Overload

• Massive Iron Deposit Assessment

V. Clinical Trials to Assess Transfusion-Related Outcomes

• Effects of Transfusion of Older Red Blood Cells on Patients With Hemoglobinopathies

VI. Clinical Trials of Treatments for Leg Ulcers

• Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders

VII. Clinical Trials of Nutritional/Dietary Supplements

• Zinc and Diabetes in Patients With Thalassemia: a Pilot Study

VIII. Observational Studies of Patients with Thalassemia

• Long Term Outcomes in β Thalassemia Major

• Reproductive Capacity and Iron Burden in Thalassemia

• Genes Influencing Iron Overload State

• Natural History of Sickle Cell Disease and Other Hemolytic Disorders

• Use of a Mobile-based App to Record Pain Levels

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

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

: Uncategorized