drugset / Trial / NCT02013375

SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD

NCT02013375

Phase 2 Terminated 2 enrolled Damiano Rondelli, MD
NaSingle-groupOpen-labelTreatment

Summary

Related donor stem cell transplantation using the alemtuzumab/ TBI platform has been shown to be a safe strategy to cure severe sickle cell disease. However, due to a lack of suitable donors, many patients cannot benefit from this strategy. Alternative donor sources are desperately needed to fill this gap. Nearly all patients will have a haploidentical family member who would be able to donate. The use of post transplantation cyclophosphamide has greatly improved the outcome of haploidentical stem cell transplantation. The investigators propose to combine this with alemtuzumab/TBI conditioning.

Timeline

Start
2014-04-10
Primary completion
2014-05-31
Completion
2018-09-07

Drugs

EvaluationDrugModalityDoseRoute
Subject Alemtuzumab Monoclonal antibody 0.3 mg/kg Intravenous
Subject Cyclophosphamide Other / unclassified 50 mg/kg Intravenous
Subject Sirolimus Small molecule 4 mg Oral

Indications