HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age3+
SponsorChildren's Hospital Medical Center, Cincinnati

About this trial

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.

Eligibility criteria

Qualifiers

Patients must have a diagnosis of Fanconi anemia

Platelet count <20 x 109/L or platelet transfusion dependence*

ANC <1000 x 109/L

Hgb <8 gm/dl or red cell transfusion dependence*

Disqualifiers

Active CNS leukemia

Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.

Active uncontrolled viral, bacterial or fungal infection

Patient seropositive for HIV-I/II; HTLV -I/II

Trial design

Treatments tested in this trial

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

Treatment groups

70 Participants
are divided into 3 treatment groups

Sponsors and collaborators

Children's Hospital Medical Center, Cincinnati

Lead sponsor

Memorial Sloan Kettering Cancer Center

Collaborator

Fred Hutchinson Cancer Center

Collaborator