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Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

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ClinicalTrials.gov Identifier: NCT01071239
Recruitment Status : Completed
First Posted : February 19, 2010
Last Update Posted : October 25, 2017
Memorial Sloan Kettering Cancer Center
Information provided by (Responsible Party):
David A. Margolis, Medical College of Wisconsin

Brief 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.

Condition or disease Intervention/treatment Phase
Fanconi Anemia Device: CliniMACs device Phase 2

Detailed Description:
We are currently recruiting patients.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
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
Study Start Date : April 2009
Primary Completion Date : August 30, 2016
Study Completion Date : August 30, 2016

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. 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 :
  1. 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 ]

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.

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

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 Posted: February 19, 2010    Key Record Dates
Last Update Posted: October 25, 2017
Last Verified: October 2017

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