drugset / Trial / NCT01499888

Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)

NCT01499888

Phase 1/2 Active not recruiting 45 enrolled University of Illinois at Chicago
NaSingle-groupOpen-labelTreatment

Summary

The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy in patients with aggressive sickle cell disease who are not candidates for or experienced complications from hydroxyurea therapy. Fully HLA matched siblings will be used as donors for hematopoietic stem cells to reduce the risk of morbidity and mortality in this cohort of patients.

Timeline

Start
2011-11-11
Primary completion
2028-09-23
Completion
2029-10

Drugs

EvaluationDrugModalityDoseRoute
Background Alemtuzumab Monoclonal antibody 1 mg/kg Intravenous
Background Sirolimus Small molecule 4 mg

Indications