drugset / Trial / NCT02143830
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Phase 2
Recruiting
70 enrolled
Children's Hospital Medical Center, Cincinnati
Fred Hutchinson Cancer Center · collabMemorial Sloan Kettering Cancer Center · collab
Non-randomizedParallel-groupOpen-labelTreatment
Summary
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
Timeline
- Start
- 2014-04
- Primary completion
- 2026-12
- Completion
- 2028-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Busulfan | Small molecule | — | Intravenous |
| Subject | Peripheral blood stem cells | Cell therapy | — | — |
| Background | Cyclophosphamide | Other / unclassified | — | Intravenous |
| Background | Fludarabine | Small molecule | — | Intravenous |
| Background | G-CSF | Unknown | — | — |
| Background | Thymoglobulin | Protein / enzyme biologic | — | — |