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Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

This study is ongoing, but not recruiting participants.
Memorial Sloan Kettering Cancer Center
Information provided by (Responsible Party):
David A. Margolis, Medical College of Wisconsin Identifier:
First received: May 20, 2009
Last updated: April 5, 2016
Last verified: April 2016
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.

Condition Intervention Phase
Fanconi Anemia
Device: CliniMACs device
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Resource links provided by NLM:

Further study details as provided by Medical College of Wisconsin:

Primary Outcome Measures:
  • To measure the incidence and quality of engraftment and hematopoietic reconstitution. [ Time Frame: 1, 3, 6 and 12 months post transplant date ]

Secondary Outcome Measures:
  • The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD [ Time Frame: weekly for the first 30 days and then 3, 6, and 12 months post transplant date ]

Estimated Enrollment: 25
Study Start Date: April 2009
Estimated Study Completion Date: May 2019
Estimated Primary Completion Date: May 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Bone marrow processing
Bone Marrow processing using the CliniMACs device
Device: CliniMACs device
Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device
Other Name: Milteny Biotec CliniMACS device

Detailed Description:
We are currently recruiting patients.

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT01071239

United States, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Medical College of Wisconsin
Memorial Sloan Kettering Cancer Center
Principal Investigator: David A Margolis, MD Medical College of Wisconsin
  More Information

Responsible Party: David A. Margolis, Professor of Pediatrics and BMT Program Director, Medical College of Wisconsin Identifier: NCT01071239     History of Changes
Other Study ID Numbers: FA 08/89
Study First Received: May 20, 2009
Last Updated: April 5, 2016

Keywords provided by Medical College of Wisconsin:
Fanconi Anemia
Hematopoietic stem cell transplant

Additional relevant MeSH terms:
Fanconi Anemia
Fanconi Syndrome
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors processed this record on April 28, 2017