drugset / Trial / NCT02678143

Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease

NCT02678143 ↗

Phase 1 Terminated 1 enrolled Washington University School of Medicine
NaSingle-groupOpen-labelTreatment

Summary

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.

Timeline

Start
2016-04-26
Primary completion
2020-04-03
Completion
2021-11-16

Outcome

Outcome not reported

Stopped: “Closed early due to competing studies”

Drugs

EvaluationDrugModalityDoseRoute
Background Alemtuzumab Monoclonal antibody — —
Background Cyclophosphamide Other / unclassified — —
Background Fludarabine Small molecule — —
Background Mycophenolate Mofetil Small molecule — —
Background Sirolimus Small molecule — —

Indications