Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease
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.
Timeline
- Start
- 2026-09
- Primary completion
- 2034-09
- Completion
- 2035-09
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | Abatacept | Other / unclassified | — | — |
| Background | Alemtuzumab | Monoclonal antibody | — | — |
| Background | Azathioprine | Small molecule | — | — |
| Background | Cyclophosphamide | Other / unclassified | 50 mg | — |
| Background | Hydroxyurea | Other / unclassified | — | — |
| Background | Sirolimus | Small molecule | — | — |
| Background | Thiotepa | Other / unclassified | — | — |
| Background | filgrastim | Protein / enzyme biologic | — | — |