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Reduced Intensity Conditioning for Haploidentical Bone Marrow Transplantation in Patients with Symptomatic Sickle Cell Disease

Study on the Safety and Effectiveness of an Investigational Haploidentical Transplant for Severe Sickle Cell Disease

Recruiting
5-45 years
All
Phase N/A

This study investigates the safety and effectiveness of a new type of transplant for people with Severe Sickle Cell Disease (SCD). The transplant is called a haploidentical transplant, which means it comes from a donor who is a half match genetically, like a parent or sibling. The purpose is to determine if this type of transplant can be a treatment option for SCD.

Participants in the study will undergo a procedure where they receive bone marrow from a related donor who is a half match. The study involves monitoring the participants for any side effects and measuring the success of the transplant in treating SCD. A haploidentical transplant involves using bone marrow from a donor who shares some, but not all, of the genetic markers with the recipient.

  • Who can participate: Participants must have sickle cell anemia and a first-degree relative willing to donate bone marrow. They must not have an HLA-matched sibling donor, uncontrolled infections, or HIV.
  • Study details: Participants will receive a bone marrow transplant from a half-matched relative. This involves a medical procedure followed by monitoring to assess the transplant's effectiveness and safety. No placebo is used in this study.
Study details
    sickle cell disease

TX8873

19 February 2024

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