NCT01071239

Hematopoietic Stem Cell Transplant for Fanconi Anemia

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Medical College of Wisconsin · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Fanconi Anemia

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2009-04-01
• Primary completion: 2016-08-30
• Completion: 2016-08-30
• First posted: 2010-02-19
• Last updated: 2019-09-11

Locations

1 site across 1 country: United States