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
Overview
- Phase
- Phase 2
- Intervention
- CliniMACs device
- Conditions
- Fanconi Anemia
- Sponsor
- Medical College of Wisconsin
- Enrollment
- 1
- Locations
- 1
- Primary Endpoint
- To measure the incidence and quality of engraftment and hematopoietic reconstitution.
- Status
- Completed
- Last Updated
- 6 years ago
Overview
Brief 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.
Investigators
David A. Margolis
Professor of Pediatrics and BMT Program Director
Medical College of Wisconsin
Eligibility Criteria
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.
Arms & Interventions
Bone marrow processing
Bone Marrow processing using the CliniMACs device
Intervention: CliniMACs device
Bone marrow processing
Bone Marrow processing using the CliniMACs device
Intervention: Busulfan
Bone marrow processing
Bone Marrow processing using the CliniMACs device
Intervention: Fludarabine
Bone marrow processing
Bone Marrow processing using the CliniMACs device
Intervention: Cyclophosphamide
Bone marrow processing
Bone Marrow processing using the CliniMACs device
Intervention: ATG
Outcomes
Primary Outcomes
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
Time Frame: 1, 3, 6 and 12 months post transplant date
To measure the incidence and quality of engraftment and hematopoietic reconstitution.
Secondary Outcomes
- The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD(weekly for the first 30 days and then 3, 6, and 12 months post transplant date)