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Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

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.
 
ClinicalTrials.gov Identifier: NCT01071239
Recruitment Status : Completed
First Posted : February 19, 2010
Last Update Posted : September 11, 2019
Sponsor:
Collaborator:
Memorial Sloan Kettering Cancer Center
Information provided by (Responsible Party):
David A. Margolis, Medical College of Wisconsin

Tracking Information
First Submitted Date  ICMJE May 20, 2009
First Posted Date  ICMJE February 19, 2010
Last Update Posted Date September 11, 2019
Study Start Date  ICMJE April 2009
Actual Primary Completion Date August 30, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 3, 2019)
To measure the incidence and quality of engraftment and hematopoietic reconstitution. [ Time Frame: 1, 3, 6 and 12 months post transplant date ]
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
Original Primary Outcome Measures  ICMJE
 (submitted: February 18, 2010)
To measure the incidence and quality of engraftment and hematopoietic reconstitution. [ Time Frame: 1, 3, 6 and 12 months post transplant date ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 3, 2019)
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 ]
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
Original Secondary Outcome Measures  ICMJE
 (submitted: February 18, 2010)
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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Hematopoietic Stem Cell Transplant for Fanconi Anemia
Official Title  ICMJE 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
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.
Detailed Description We are currently recruiting patients.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fanconi Anemia
Intervention  ICMJE
  • 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
  • Drug: Busulfan
    Chemotherapy administered as a part of the HSCT conditioning regimen.
  • Drug: Fludarabine
    Chemotherapy administered as a part of the HSCT conditioning regimen.
  • Drug: Cyclophosphamide
    Chemotherapy administered as a part of the HSCT conditioning regimen.
  • Drug: ATG
    Chemotherapy administered as a part of the HSCT conditioning regimen.
Study Arms  ICMJE Experimental: Bone marrow processing
Bone Marrow processing using the CliniMACs device
Interventions:
  • Device: CliniMACs device
  • Drug: Busulfan
  • Drug: Fludarabine
  • Drug: Cyclophosphamide
  • Drug: ATG
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 21, 2018)
1
Original Estimated Enrollment  ICMJE
 (submitted: February 18, 2010)
25
Actual Study Completion Date  ICMJE August 30, 2016
Actual Primary Completion Date August 30, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01071239
Other Study ID Numbers  ICMJE FA 08/89
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party David A. Margolis, Medical College of Wisconsin
Study Sponsor  ICMJE Medical College of Wisconsin
Collaborators  ICMJE Memorial Sloan Kettering Cancer Center
Investigators  ICMJE
Principal Investigator: David A Margolis, MD Medical College of Wisconsin
PRS Account Medical College of Wisconsin
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP