Clinical Trial

Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease

Recruiting Phase 2
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Summary
The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the goal to improve the GVHD-free rejection-free survival (GRFS) to greater than 90% after haploidentical donor HCT in children and young adults with SCD. Primary Objective: \- To assess the GVHD-free and rejection free survival (GRFS) after haploidentical donor HCT in children and young adults with SCD. Secondary Objectives: * Assess the overall survival (OS) and disease-free survival (DFS) after haploidentical donor HCT for SCD. * Estimate incidence and severity of acute and chronic GVHD after haploidentical donor HCT for SCD. * Assess the neutrophil and platelet engraftment kinetics after haploidentical donor HCT for SCD.
Protocol Amendment History 1 change
critical Recruitment opened 2026-07-24
Trial Details
NCT Number NCT07616154
Lead Sponsor St. Jude Children's Research Hospital
Conditions Sickle Cell Disease
Enrollment 45 participants
Start Date 2026-09
Primary Completion 2034-09 (estimated)
Study Completion 2035-09 (estimated)
Updated on ClinicalTrials.gov 2026-07-23