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Hematopoietic Stem Cell Transplant for Fanconi Anemia

NCT01071239 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2019-09-11

No results posted yet for this study

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.

Conditions

  • Fanconi Anemia

Interventions

DEVICE

CliniMACs device

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

Sponsors & Collaborators

Principal Investigators

  • David A Margolis, MD · Medical College of Wisconsin

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-04-30
Primary Completion
2016-08-30
Completion
2016-08-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01071239 on ClinicalTrials.gov