Clinical Trial: Multicenter Transplant Study for Fanconi Anemia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a

Brief Summary: The trial proposed is a multicenter treatment protocol designed to examine transplant related events in patients with Fanconi anemia who lack matched sib donors have severe aplastic anemia (SAA), or myelodysplastic syndrome(MDS) or acute myelogenous leukemia (AML).

Detailed Summary: The trial proposed is a single arm phase II multicenter 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 ATG (Anti-thymocyte globulin)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 (HLA = human leukocyte antigen) identical donors using stem cell transplants derived from HLA-compatible unrelated donors or HLA haplotype-mismatched related donors using Miltenyi's CliniMACS.
Sponsor: Children's Hospital Medical Center, Cincinnati

Current Primary Outcome: The primary objectives are: The incidence and quality of engraftment and hematopoietic reconstitution. The incidence of early transplant related mortality The incidence and severity of acute GvHD (graft versus host disease) and chronic GvHD. [ Time Frame: 1-5 years ]

Original Primary Outcome: The primary objectives of this trial are as follows: The incidence and quality of engraftment and hematopoietic reconstitution. The incidence of early transplant related mortality The incidence and severity of acute GvHD and chronic GVHD. [ Time Frame: 1-5 years ]

Current Secondary Outcome: The incidence of overall survival and disease free survival over time. [ Time Frame: 5 Years ]

Original Secondary Outcome: Same as current

Information By: Children's Hospital Medical Center, Cincinnati

Dates:
Date Received: March 5, 2010
Date Started: October 2009
Date Completion:
Last Updated: June 28, 2016
Last Verified: June 2016