Hematopoietic Stem Cell Transplant for Fanconi Anemia

NCT ID: NCT01071239

Last Updated: 2019-09-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-04-30

Study Completion Date

2016-08-30

Brief Summary

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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

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We are currently recruiting patients.

Conditions

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Fanconi Anemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Bone marrow processing

Bone Marrow processing using the CliniMACs device

Group Type EXPERIMENTAL

CliniMACs device

Intervention Type DEVICE

Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device

Busulfan

Intervention Type DRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Fludarabine

Intervention Type DRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Cyclophosphamide

Intervention Type DRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

ATG

Intervention Type DRUG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Interventions

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CliniMACs device

Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device

Intervention Type DEVICE

Busulfan

Chemotherapy administered as a part of the HSCT conditioning regimen.

Intervention Type DRUG

Fludarabine

Chemotherapy administered as a part of the HSCT conditioning regimen.

Intervention Type DRUG

Cyclophosphamide

Chemotherapy administered as a part of the HSCT conditioning regimen.

Intervention Type DRUG

ATG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Intervention Type DRUG

Other Intervention Names

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Milteny Biotec CliniMACS device

Eligibility Criteria

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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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Memorial Sloan Kettering Cancer Center

OTHER

Sponsor Role collaborator

Medical College of Wisconsin

OTHER

Sponsor Role lead

Responsible Party

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David A. Margolis

Professor of Pediatrics and BMT Program Director

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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David A Margolis, MD

Role: PRINCIPAL_INVESTIGATOR

Medical College of Wisconsin

Locations

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Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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FA 08/89

Identifier Type: -

Identifier Source: org_study_id

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