NCT02143830 HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
| NCT ID | NCT02143830 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Children's Hospital Medical Center, Cincinnati |
| Condition | Fanconi Anemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 70 participants |
| Start Date | 2014-04 |
| Primary Completion | 2026-12 |
Trial Parameters
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Brief Summary
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
Eligibility Criteria
Inclusion Criteria: * Patients must have a diagnosis of Fanconi anemia * Patients must have one of the following hematologic diagnoses: 1. Severe Aplastic Anemia (SAA), with bone marrow cellularity of \<25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following features: 1. Platelet count \<20 x 109/L or platelet transfusion dependence\* 2. ANC \<1000 x 109/L 3. Hgb \<8 gm/dl or red cell transfusion dependence\* 2. Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification 3. Acute Myelogenous Leukemia (untreated, in remission or with refractory or relapsed disease) * Donors will be either human leukocyte antigen (HLA) compatible unrelated or HLA-genotypically matched related donors (no fully matched sibling donor). * Patients and donors may be of either gender or any ethnic background. * Patients must have a Karnofsky adult, or Lansky pediatric performance scale status \> 70%. * Patients must have adequate physical function measured by: 1. Cardiac: asym