Actively Recruiting
A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor Using Risk-Adjusted Chemotherapy With Busulfan, Cyclophosphamide, and Fludarabine
Led by Children's Hospital Medical Center, Cincinnati · Updated on 2025-11-12
70
Participants Needed
3
Research Sites
104 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
Researchers are studying a new approach to stem cell transplant treatment for patients with Fanconi Anemia (FA), a condition that causes severe marrow failure, myelodysplastic syndrome, or acute myelogenous leukemia. This phase II trial aims to find out if using lower doses of busulfan and removing cyclosporine can reduce transplant-related side effects while maintaining effective disease control. Patients will receive a risk-adjusted chemotherapy regimen combined with a specialized stem cell transplant from mismatched related or matched unrelated donors. The treatment includes three groups based on age and disease status. Younger patients with marrow aplasia or single lineage cytopenias receive standard busulfan doses plus cyclophosphamide, fludarabine, rabbit ATG before transplant, and G-CSF after transplant. Patients with myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML) receive a higher busulfan dose with similar supporting drugs and procedures. Older patients get a slightly reduced busulfan dose. All patients receive peripheral blood stem cells that are T-cell depleted using a CD34 selection method via the Miltenyi CliniMACS device. Cyclosporine is not used to prevent graft-versus-host disease (GVHD). Participants will be closely monitored over several years, with the main measurement being graft failure or rejection at five years post-transplant. Additional assessments include tracking severe complications and mortality up to two years after transplant. The study involves detailed pre-transplant evaluations such as heart, liver, kidney, and lung function tests, and ongoing evaluations during and after treatment. Enrollment includes children and adults starting from 3 months old, and all patients must provide informed consent to take part.
CONDITIONS
Brief 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 severe marrow failure (transfusion dependent), myelodysplastic syndrome, or acute myelogenous leukemia
- Donors must be HLA-compatible unrelated or HLA-genotypically matched related (no fully matched sibling donor)
- Patients and donors may be any gender or ethnic background
- Patients must have a Karnofsky adult or Lansky pediatric performance status above 70%
- Patients must have adequate heart, liver, kidney, and lung function as specified
- Female patients and donors must not be pregnant or breastfeeding and must agree to pregnancy testing and prevention during the study
- Patients must be willing to participate and sign informed consent
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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 days of chemotherapy infusions plus stem cell transplant procedure
Participants receive risk-adjusted chemotherapy with busulfan, cyclophosphamide, and fludarabine, followed by a CD34+ selected T-cell depleted peripheral blood stem cell transplant. Rabbit ATG is given prior to transplant and G-CSF is administered post-transplant to promote engraftment.
Multiple visits during the 4-day chemotherapy and transplant period
Duration - Up to 5 years
Participants are monitored for graft failure, rejection, and post-transplant severe morbidity and mortality over several years after the transplant.
Regular follow-up visits over 2 to 5 years post-transplant
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
S
Sara Loveless, RN
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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