Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)

September 3, 2019 updated by: David A. Margolis, Medical College of Wisconsin

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

We are currently recruiting patients.

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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 Names:
  • 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
1, 3, 6 and 12 months post transplant date

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
weekly for the first 30 days and then 3, 6, and 12 months post transplant date

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medical College of Wisconsin

Collaborators

Memorial Sloan Kettering Cancer Center

Investigators

  • Principal Investigator: David A Margolis, MD, Medical College of Wisconsin

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2009

Primary Completion (Actual)

August 30, 2016

Study Completion (Actual)

August 30, 2016

Study Registration Dates

First Submitted

May 20, 2009

First Submitted That Met QC Criteria

February 18, 2010

First Posted (Estimate)

February 19, 2010

Study Record Updates

Last Update Posted (Actual)

September 11, 2019

Last Update Submitted That Met QC Criteria

September 3, 2019

Last Verified

September 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FA 08/89

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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