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Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

This study is ongoing, but not recruiting participants.
ClinicalTrials.gov Identifier:
First Posted: February 19, 2010
Last Update Posted: April 6, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Memorial Sloan Kettering Cancer Center
Information provided by (Responsible Party):
David A. Margolis, Medical College of Wisconsin
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: None (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 David A. Margolis, 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.

Information from the National Library of Medicine

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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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
ClinicalTrials.gov Identifier: NCT01071239     History of Changes
Other Study ID Numbers: FA 08/89
First Submitted: May 20, 2009
First Posted: February 19, 2010
Last Update Posted: April 6, 2016
Last Verified: April 2016

Keywords provided by David A. Margolis, 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