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Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

Primary Purpose

Fanconi Anemia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
CliniMACs device
Busulfan
Fludarabine
Cyclophosphamide
ATG
Sponsored by
Medical College of Wisconsin
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fanconi Anemia focused on measuring Fanconi Anemia, Hematopoietic stem cell transplant

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Medical College of Wisconsin

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Bone marrow processing

Arm Description

Bone Marrow processing using the CliniMACs device

Outcomes

Primary Outcome Measures

To measure the incidence and quality of engraftment and hematopoietic reconstitution.
To measure the incidence and quality of engraftment and hematopoietic reconstitution.

Secondary Outcome Measures

The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD

Full Information

First Posted
May 20, 2009
Last Updated
September 3, 2019
Sponsor
Medical College of Wisconsin
Collaborators
Memorial Sloan Kettering Cancer Center
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1. Study Identification

Unique Protocol Identification Number
NCT01071239
Brief Title
Hematopoietic Stem Cell Transplant for Fanconi Anemia
Acronym
FA
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 Type
Interventional

2. Study Status

Record Verification Date
September 2019
Overall Recruitment Status
Completed
Study Start Date
April 2009 (undefined)
Primary Completion Date
August 30, 2016 (Actual)
Study Completion Date
August 30, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Medical College of Wisconsin
Collaborators
Memorial Sloan Kettering Cancer Center

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi Anemia
Keywords
Fanconi Anemia, Hematopoietic stem cell transplant

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Bone marrow processing
Arm Type
Experimental
Arm Description
Bone Marrow processing using the CliniMACs device
Intervention Type
Device
Intervention Name(s)
CliniMACs device
Other Intervention Name(s)
Milteny Biotec CliniMACS device
Intervention Description
Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device
Intervention Type
Drug
Intervention Name(s)
Busulfan
Intervention Description
Chemotherapy administered as a part of the HSCT conditioning regimen.
Intervention Type
Drug
Intervention Name(s)
Fludarabine
Intervention Description
Chemotherapy administered as a part of the HSCT conditioning regimen.
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide
Intervention Description
Chemotherapy administered as a part of the HSCT conditioning regimen.
Intervention Type
Drug
Intervention Name(s)
ATG
Intervention Description
Chemotherapy administered as a part of the HSCT conditioning regimen.
Primary Outcome Measure Information:
Title
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
Description
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
Time Frame
1, 3, 6 and 12 months post transplant date
Secondary Outcome Measure Information:
Title
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD
Description
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

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
David A Margolis, MD
Organizational Affiliation
Medical College of Wisconsin
Official's Role
Principal Investigator
Facility Information:
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States

12. IPD Sharing Statement

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Hematopoietic Stem Cell Transplant for Fanconi Anemia

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