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

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

Category:

Supportive Hematological Disorders Studies

Diseases Treated:

Sickle cell anemia

Eligibility Overview:

This is a non-therapeutic clinical trial that is only open to St. Jude patients.

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

Description

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 will use 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 will be asked to donate stem cells and have them collected by a procedure known as bone marrow aspiration.

Objectives

  • Researchers hope to use knowledge derived from this study to develop a treatment for use in future patients who have sickle cell disease or thalassemia due to a defective gene.

Eligibility

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

Only current St. Jude patients are eligible.

Principal Investigator

Arthur Nienhuis, MD

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.