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
Brief description of study.
Detailed description of study
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: sickle cell disease
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Age: 5 years - 45 years
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Gender: All
Inclusion Criteria
Adequate physical function
Participants must have a first-degree related HLA-haploidentical related donor who is willing and able to donate bone marrow
Participants with sickle cell anemia
Exclusion Criteria
Participants who have an HLA-matched sibling who is able and willing to donate bone marrow. Patients with a HLA-matched unrelated donor are not excluded.
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 disease, requiring active medical management (immunosuppressive therapy or chemotherapy), which, in the judgment of the local Principal Investigator, indicates that the patient could not tolerate transplantation
Females of child bearing potential (to include all female participants > 10 years of age, unless postmenopausal for a minimum of 1 year before the time of consent or surgically sterilized), who do not agree to practice two (2) effective methods of contraception at the same time, or do not agree to practice true abstinence when this is in line with the preferred and usual lifestyle of the subject, from the time of signing of informed consent through 12 months post-transplant.
Males (even if surgical sterilized) who do not agree to practice effective barrier contraception, or who do not agree to practice true abstinence from the time of signing informed consent through 12 months post-transplant
Anti-donor specific HLA antibodies: Positive anti-donor HLA antibody is defined as a positive crossmatch test of any titer (by complement-dependent cytotoxicity or flow cytometric testing) or the presence of anti-donor HLA antibody to the high expression loci HLA-A, -B, -C, or ?DRB1 with mean fluorescence intensity >3000 by solid phaseimmunoassay. This will be measured before the final donor selection, and at least 180 days or less before HCT
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.
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