HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Recruiting Phase 2 Interventional Study
Fanconi Anemia Severe Marrow Failure Myelodysplastic Syndrome (MDS) Acute Myelogenous Leukemia (AML)
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
3 Months and older
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
70 (estimated)
Sponsor
Children's Hospital Medical Center, Cincinnati · Other
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About This Trial
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 cyclophos…
Trial Locations
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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: …
Contacts

Jamie Wilhelm

(513)803-1102

Jamie.Wilhelm@cchmc.org

CONTACT

Sara Loveless, RN

(513)803-7656

Sara.Loveless@cchmc.org

CONTACT