NCT00453388

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia

Nonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding Study

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This phase II trial studies how well total-body irradiation (TBI) works when given together with fludarabine phosphate and cyclophosphamide followed by donor bone marrow transplant, mycophenolate mofetil, and cyclosporine in treating patients with Fanconi anemia (FA). Giving low doses of chemotherapy, such as fludarabine phosphate and cyclophosphamide, and TBI before or after a donor bone marrow transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.

Detailed description

PRIMARY OBJECTIVES: I. Identify doses of total-body irradiation (TBI) that lead to sufficient probability of donor engraftment (\> 5% donor cluster of differentiation \[CD\]3 chimerism) by day +200. II. Evaluate the probability of severe acute graft-versus-host disease. SECONDARY OBJECTIVES: I. Evaluate the probabilities of overall survival, regimen-related toxicity (RRT), and recurrent hematopoietic malignancy in those patients with a prior underlying history of such. II. Examine the degree to which mixed chimerism provides for amelioration of symptoms (i.e., infections due to neutropenia, hemorrhage due to thrombocytopenia) associated with bone marrow failure. III. Determine if the FA complementation group and % initial mosaicism predict engraftment and RRT outcomes. OUTLINE: Patients are assigned to 1 of 4 treatment arms. NOTE: Patients no longer receive pre-transplant cyclophosphamide as of February 2009. After completion of study treatment, patients are followed up at 6 months and then annually thereafter.

Conditions

• Acute Myeloid Leukemia in Remission
• de Novo Myelodysplastic Syndrome
• Fanconi Anemia
• Previously Treated Myelodysplastic Syndrome

Interventions

Type	Name	Description
PROCEDURE	Allogeneic Bone Marrow Transplantation	Undergo allogeneic bone marrow transplant
DRUG	Cyclophosphamide	Given IV
DRUG	Cyclosporine	Given IV or PO
DRUG	Fludarabine Phosphate	Given IV
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Mycophenolate Mofetil	Given PO
PROCEDURE	Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation	Undergo allogeneic stem cell transplant
RADIATION	Total-Body Irradiation	Undergo TBI

Timeline

• Start date: 2007-02-01
• Primary completion: 2013-06-01
• Completion: 2013-06-01
• First posted: 2007-03-28
• Last updated: 2020-01-29
• Results posted: 2017-05-24

Locations

5 sites across 2 countries: United States, Brazil