BMR: Bone Marrow Collection for Sickle Cell and Thalassemia Research

Bone Marrow for Hemoglobinopathy Research

Category:

Supportive Hematological Disorders Studies

Diseases Treated:

Sickle cell anemia, thalassemia

Eligibility Overview:

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

  • At least 2 years old
  • Diagnosed with sickle cell anemia or thalassemia
  • No active disease, including painful crisis or active infection
  • Adequate blood counts
  1. These studies will evaluate the potential of retroviral vector mediated gene transfer, gene editing (via CRISPR, Talen, zinc finger, or other novel modifications), or drug treatment (epigenetic modifier drugs), to correct the pathophysiology of sickle cell anemia and beta thalassemia.

    CD34+ cells purified from bone marrow of research participants with a sickle cell syndrome will undergo genetic editing, drug treatment or be transduced with retroviral vectors to enhance gene expression and insulate regulatory elements from cellular genes at or near the integration sites.

    Potential participants include adults at least 18 years old or children between the ages of 2 and 17 who are scheduled to be sedated or undergo general anesthesia for an unrelated diagnostic or surgical procedure.

    Primary Objective

    • To develop retroviral vector mediated gene transfer as a potentially curative therapy for sickle cell anemia and beta thalassemia
    • To develop a gene editing strategy which elevates the expression of fetal hemoglobin to potentially curative level for sickle cell anemia and β-thalassemia
    • To develop a drug treatment strategy which elevates the expression of fetal hemoglobin to a potentially curative level for sickle cell anemia and β-thalassemia

    Eligibility Criteria

    Inclusion criteria include:

    • At least 2 years old
    • Homozygous S/S disease or doubly heterozygous for S and beta thalassemia OR
    • HbE beta thalassemia or homozygous (severe) beta thalassemia
    • Adequate blood counts

    Exclusion criteria include:

    • Pregnancy
    • Active sickle cell disease (pain crises, acute chest syndrome, cerebrovascular events or active infection)

    Only current St. Jude patients are eligible.

    Study Sites

    St. Jude Children’s Research Hospital, Memphis, Tennessee

  2. 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 beta thalassemia. Stem cells are able to become any type of blood cell.  They usually live in the bone marrow. 

    If you participate in this study, you will be asked to donate stem cells. Researchers will collect your cells by taking a sample of your liquid marrow through a needle. This procedure is known as bone marrow aspiration.

    Purpose of this clinical trial

    The main goal of this study is to explore new treatments for sickle cell disease and thalassemia.

    Eligibility overview

    • At least 2 years old
    • Diagnosed with sickle cell anemia or thalassemia
    • No active disease, including painful crisis or active infection
    • Adequate blood counts
  3. BMR Quick View
    Sponsor St. Jude Children’s Research Hospital
    ClinicalTrials.gov identifier NCT00669305
    Trial Start Date May 2008
    Estimated Enrollment 28
    Study Type Observational
    Conditions Sickle cell anemia, thalassemia
    Ages 2 years or older
    Principal Investigator Mitch Weiss, MD, PhD
    Study Sites St. Jude Children’s Research Hospital
    For a consultation or to discuss BMR St. Jude Physician/Patient Referral Office
    1-888-226-4343
    referralinfo@stjude.org

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.