Fanconi Anemia
Conditions
Keywords
Fanconi Anemia, Hematopoietic stem cell transplant
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.
Interventions
DEVICECliniMACs device
Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device
DRUGBusulfan
Chemotherapy administered as a part of the HSCT conditioning regimen.
DRUGFludarabine
Chemotherapy administered as a part of the HSCT conditioning regimen.
DRUGCyclophosphamide
Chemotherapy administered as a part of the HSCT conditioning regimen.
DRUGATG
Chemotherapy administered as a part of the HSCT conditioning regimen.
Sponsors
Memorial Sloan Kettering Cancer Center
Medical College of Wisconsin
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To measure the incidence and quality of engraftment and hematopoietic reconstitution. | 1, 3, 6 and 12 months post transplant date | To measure the incidence and quality of engraftment and hematopoietic reconstitution. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD |
Countries
United States
Outcome results
None listed