Status: Recruiting
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
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.
Participants needed: 70
Trial details
Phase: Phase 2Age: 3+Biological sex: AllType: InterventionalSponsor: Children's Hospital Medical Center, CincinnatiUpdated: Nov 12, 2025Locations: 3
Eligibility criteria
Patients must have a diagnosis of Fanconi anemia [+14]
Active CNS leukemia [+3]