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.

Trial Details

NCT ID

NCT02143830

Phase

PHASE2

Sponsor

Children's Hospital Medical Center, Cincinnati

Status

RECRUITING

Cancer Type

Leukemia

Interventions

Busulfan
Cyclophosphamide
Fludarabine
rabbit ATG
G-CSF
Peripheral blood stem cell

Locations (sample)

New York, New York, United States|40.71427,-74.00597
Cincinnati, Ohio, United States|39.12711,-84.51439
Seattle, Washington, United States|47.60621,-122.33207

Key Eligibility Criteria

Patients must have a diagnosis of Fanconi anemia
Patients must have one of the following hematologic diagnoses:
Severe Aplastic Anemia (SAA), with bone marrow cellularity of \<25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following f…
Platelet count \<20 x 109/L or platelet transfusion dependence\*

For full eligibility, visit ClinicalTrials.gov.

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