drugset / Trial / NCT03214354

Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

NCT03214354

Phase 2 Recruiting 12 enrolled University of Calgary
NaSingle-groupOpen-labelTreatment

Summary

The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.

Timeline

Start
2017-07-05
Primary completion
2028-07
Completion
2028-07

Drugs

EvaluationDrugModalityDoseRoute
Background Alemtuzumab Monoclonal antibody 0.2 mg/kg Subcutaneous
Background Sirolimus Small molecule