Overview

This trial is active, not recruiting.

Condition fanconi anemia
Treatment clinimacs device
Phase phase 2
Sponsor Medical College of Wisconsin
Collaborator Memorial Sloan Kettering Cancer Center
Start date April 2009
End date May 2018
Trial size 25 participants
Trial identifier NCT01071239, FA 08/89

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Bone Marrow processing using the CliniMACs device
clinimacs device Milteny Biotec CliniMACS device
Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device

Primary Outcomes

Measure
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
time frame: 1, 3, 6 and 12 months post transplant date

Secondary Outcomes

Measure
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

Eligibility Criteria

Male or female participants of any age.

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.

Additional Information

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
Principal investigator David A Margolis, MD
Description We are currently recruiting patients.
Trial information was received from ClinicalTrials.gov and was last updated in September 2015.
Information provided to ClinicalTrials.gov by Medical College of Wisconsin.