Actively Recruiting
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Led by Children's Hospital Medical Center, Cincinnati · Updated on 2025-11-12
70
Participants Needed
3
Research Sites
765 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
M
Memorial Sloan Kettering Cancer Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must have a diagnosis of Fanconi anemia
- Patients must have one of the following hematologic diagnoses: severe aplastic anemia with bone marrow cellularity <25% or severe isolated single lineage cytopenia with platelet count <20 x 10^9/L or platelet transfusion dependence, ANC <1000 x 10^9/L, or hemoglobin <8 gm/dl or red cell transfusion dependence; myelodysplastic syndrome based on WHO or IPSS classification; or acute myelogenous leukemia (untreated, in remission, or refractory/relapsed)
- Donors must be HLA-compatible unrelated or HLA-genotypically matched related donors (no fully matched sibling donor)
- Patients and donors may be any gender or ethnicity
- Karnofsky adult or Lansky pediatric performance status >70%
- Adequate physical function: cardiac function asymptomatic or LVEF >50% improving with exercise or shortening fraction >29%; hepatic function with ALT <5x upper limit of normal and total bilirubin <2.0 mg/dl; renal function with serum creatinine <1.5 mg/dl or creatinine clearance >50 ml/min/1.73 m2; pulmonary function asymptomatic or DLCO >50% predicted
- Willingness to participate and sign informed consent
- Female patients and donors not pregnant or breastfeeding at consent and willing to undergo pregnancy testing prior to transplant and avoid pregnancy during study
You will not qualify if you...
- Active central nervous system leukemia
- Female patients who are pregnant or breastfeeding
- Active uncontrolled viral, bacterial, or fungal infection
- Patient seropositive for HIV-I/II or HTLV-I/II
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10174
Completed
2
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
3
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States, 98109
Actively Recruiting
Research Team
J
Jamie Wilhelm
CONTACT
S
Sara Loveless, RN
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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