Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2013 by Medical College of Wisconsin
Sponsor:
Collaborator:
Memorial Sloan-Kettering Cancer Center
Information provided by (Responsible Party):
David A. Margolis, Medical College of Wisconsin
ClinicalTrials.gov Identifier:
NCT01071239
First received: May 20, 2009
Last updated: October 23, 2013
Last verified: October 2013
  Purpose

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: Endpoint Classification: Safety/Efficacy Study
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 ] [ Designated as safety issue: Yes ]

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 ] [ Designated as safety issue: Yes ]

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.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01071239

Contacts
Contact: David A Margolis, MD 414-955-4170 dmargoli@mcw.edu
Contact: Jennifer Brown 414-266-8914 jibrown@mcw.edu

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

No publications provided

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
Study First Received: May 20, 2009
Last Updated: October 23, 2013
Health Authority: United States: Food and Drug Administration

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

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

ClinicalTrials.gov processed this record on August 28, 2014