Status and phase
Conditions
Treatments
About
The aim of this study is to evaluate the overall safety and feasibility of using haploidentical or one antigen mismatch unrelated hematopoietic stem cell transplant (HSCT) for adult patients with severe sickle cell disease (SCD) who undergo a non-myeloablative preparative regimen consisting of total body irradiation (TBI), cyclophosphamide and alemtuzumab (and fludarabine for haplo-SCT only) and graft vs. host disease (GvHD) prophylaxis consisting of post-transplant cyclophosphamide (PT-Cy), mycophenolate mofetil (MMF), and sirolimus. The investigators anticipate that this approach will expand the donor pool and offer a safe and less toxic curative intervention.
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Recipient Inclusion Criteria:
Age greater than or equal to 19 years.
Diagnosis of sickle cell disease (HB SS, SC, or SBeta-thal(0)); confirmed by hemoglobin electrophoresis, high-performance liquid chromatography, DNA testing when necessary or both.
At high risk for disease-related morbidity or mortality, defined by having at least one of the following manifestations (A-E):
Any one of the below complications not ameliorated by hydroxyurea at the maximum tolerated dose for at least 6 months:
Availability of one antigen mismatched unrelated or haploidentical related donor
Cerebral MRI/MRA within 30 days prior to initiation of transplant conditioning. If there is clinical or radiologic evidence of a recent neurologic event (such as stroke or transient ischemic attack) subjects will be deferred for at least 6 months with repeat cerebral MRI/MRA to ensure stabilization of the neurologic event prior to proceeding to transplantation.
Ability to comprehend and willing to sign an informed consent
Recipient Exclusion Criteria:
Donor Selection:
Must be one antigen mismatched unrelated donor or first-degree relative who shares at least one HLA haplotype with the recipient.
Must not have SCD or another hemoglobinopathy.
In good health based on institutional standards.
Weight ≥ 20kg.
If donor is < 18 years old must have ability to give informed assent based on institutional standards for pediatric donors.
Able to undergo peripheral blood stem cell mobilization with G-CSF
Hemoglobin S ≤ 50%.
HIV-1&2 antibody, HTLV-I&II antibody, HBV and HCV sero-negative.
Note: When more than one donor is available, the donor with the lowest number of HLA allele mismatches will be chosen, unless there is HLA cross-match incompatibility or a medical reason to select otherwise, in which case donor selection is the responsibility of the PI, in consultation with the immunogenetics laboratory. In cases where there is more than one donor with the least degree of mismatch, donors will be selected based on the most favorable combination of (i) HLA compatibility in cross-match testing, (ii) ABO compatibility, (iii) CMV status and (iv) non-inherited maternal antigens (NIMAs) mismatching.
HLA crossmatching (in order of priority)
ABO compatibility (in order of priority)
CMV negative donor is preferred
NIMA mismatched donor is preferred
Primary purpose
Allocation
Interventional model
Masking
1 participants in 1 patient group
Loading...
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal