Hematopoietic Stem Cell Transplant for Fanconi Anemia

• Status: Completed
• Trial end date: 2016-08-30
• Target enrollment: 0
• Participant gender: All

Summary

The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically identical donors using stem cell transplants derived from (1) HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Medical College of Wisconsin
• Collaborator: Memorial Sloan Kettering Cancer Center
• Treatments: Busulfan; Cyclophosphamide; Fludarabine; Fludarabine phosphate

Criteria

Inclusion Criteria:

• Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia

• Karnofsky or Lansy performance scale > or = to 70%.

• Must have adequate cardiac, hepatic, renal and pulmonary function.

• Must have 7/8 or 8/8 available unrelated donor.

Exclusion Criteria:

• Pregnant or breastfeeding.

• Active CNS leukemic involvement

• Active uncontrolled viral, bacterial or fungal infection

• Positive for HIV.