Trial Information

Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia

OBJECTIVES:

- Decrease the conditioning-related toxicity of cyclophosphamide without decreasing the
engraftment rate to < 90% in patients undergoing allogeneic bone marrow transplantation
for Fanconi's anemia.

OUTLINE: This is a multicenter, dose-finding study of cyclophosphamide.

- Nonmyeloablative conditioning regimen: Patients receive cyclophosphamide IV on days -5
to -2.

Cohorts of 5-10 patients receive decreasing doses of cyclophosphamide until the optimal dose
(OD) is determined. The OD is defined as the dose at which ≥ 4 of 5 patients achieve
engraftment and < 1 of 10 patients experiences dose-limiting toxicity.

- Allogeneic bone marrow transplantation (BMT): Patients undergo allogeneic BMT on day 0.

- Graft-vs-host-disease (GVHD) prophylaxis: Patients receive cyclosporine orally or IV
twice daily beginning on day -1 and continuing until day 49, followed by a taper on
days 50-180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3,
6, and 11.

After completion of study treatment, patients are followed periodically for 5 years.

PROJECTED ACCRUAL: A total of 27 patients will be accrued for this study.