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

About this study

Sickle cell anemia is an inherited disease primarily affecting individuals of African American descent.  It can both shorten and reduce the quality of life by creating risk for strokes and causing chronic organ damage.

Gene therapy may potentially address the lack of available healing therapy. This study used bone marrow stem cells from patients with sickle cell disease or thalassemia. Stem cells are able to become any type of blood cell. They usually live in the bone marrow. Participants were asked to donate stem cells and have them collected by a procedure known as bone marrow aspiration.

Eligibility overview

  • Participant’s age was equal to or greater than two (2) years old
  • No active sickle cell disease
  • Adequate blood counts

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Overview

Full title:

Experimental Evaluation of the Potential to Correct the Pathophysiology of Sickle Cell Anemia or Thalassemia by Retroviral Vector Mediated Globin Gene Transfer

Study goal:

To develop a treatment for use in future patients who have sickle cell disease or thalassemia due to a defective gene

Diagnosis:

Sickle cell anemia

Age:

Equal or greater than 2 years old

Clinical trials categories:

Sickle Cell Anemia Blood Disorders Beta Thalassemia

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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