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

Study on Safety and Effectiveness of Haploidentical Transplant for Sickle Cell Disease

Recruiting
5 years - 45 years
All
Phase N/A
1 Location

Brief description of study.

We're doing this study to learn if a haploidentical transplant is safe and effective to treat SCD.

Detailed description of study

The purpose of this study is to learn if a new type of allogeneic transplant called a haploidentical transplant is safe and effective to treat SCD.
Participants will be compensated for their participation.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: sickle cell disease
  • Age: 5 years - 45 years
  • Gender: All

Inclusion Criteria
Adequate physical function
Must have a first-degree related HLA-haploidentical related donor who is willing and able to donate blood marrow
Exclusion Criteria
Participants who have an HLA-matched sibling who is able and willing to donate bone marrow
Uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment
Evidence of HIV infection or known HIV positive serology
Participants who have received a previous HCT
Participants who have participated in another clinical trial in which the patient received an investigational or off-label use of a drug or device within 3 months of enrollment
Females who are pregnant or breastfeeding
Participants with clinically significant, uncontrolled autoimmune diesease, requiring active medical management, which, in the judgment of the local Principal Investigator, indicates that the patient could not tolerate transplantation

Updated on 19 Feb 2024. Study ID: 1705650500

This study investigates the use of a haploidentical transplant, a type of allogeneic transplant, to see if it is safe and effective for treating sickle cell disease (SCD). Sickle cell disease is a condition where red blood cells are shaped like a sickle, which can cause pain and other serious problems. A haploidentical transplant involves using a donor who is a half-match to the recipient, which might offer more treatment options for patients.

Participants in the study will undergo a procedure where bone marrow is transplanted from a donor who is a close family member. This process is called a haploidentical transplant. The study will monitor the safety and effectiveness of this procedure in treating SCD. Participants will be compensated for their involvement in the study.

  • Who can participate: Adults with adequate physical function who have a first-degree relative willing to donate bone marrow can participate. Those with an HLA-matched sibling donor, uncontrolled infections, previous HCT, participation in another research study recently, pregnancy, or uncontrolled autoimmune diseases cannot join.
  • Study details: Participants will receive a haploidentical transplant from a related donor. This involves transferring bone marrow from a family member who is a partial genetic match.

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