Hematopoietic Stem Cell Transplant for Fanconi Anemia

Completed · Phase 2

Conditions studied: Fanconi Anemia

In brief

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.

Key facts

Study ID

NCT01071239

Run by

Medical College of Wisconsin

People needed

1

Starts

2009-04-01

Expected to finish

2016-08-30

Last updated by the study team

2019-09-11

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Pregnant or breastfeeding.
• Active CNS leukemic involvement
• Active uncontrolled viral, bacterial or fungal infection
• Positive for HIV.

Where it is running

• Medical College of Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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