drugset / Trial / NCT03121001
Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
NaSingle-groupOpen-labelTreatment
Summary
The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease.
Timeline
- Start
- 2017-03-20
- Primary completion
- 2029-05
- Completion
- 2029-05
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | ATG | Unknown | 2 mg/kg | Intravenous |
| Background | Cyclophosphamide | Other / unclassified | 50 mg/kg | Intravenous |
| Background | Fludarabine | Small molecule | 30 mg/m2 | Intravenous |
| Background | Mycophenolate Mofetil | Small molecule | 1 g | — |
| Background | Sirolimus | Small molecule | 5 mg | — |