Briquilimab with reduced donor stem cell transplant for Fanconi anemia
Treatments studied
Part of Blood & lymphatic, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests whether a reduced-intensity donor stem cell transplant (with briquilimab as part of the conditioning) can safely treat bone marrow failure in people with Fanconi anemia. It may help by lowering how much intensive treatment is needed before the transplant, while still helping the donor cells grow.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have Fanconi anemia confirmed by special chromosome testing and a known gene mutation
- You have bone marrow failure (low blood counts) showing up on two tests at least a month apart
- You are age 2 or older
- You have a donor matched to you on HLA: at least 5 out of 10 matches, and the donor can give cells for an apheresis procedure
- Your body systems are healthy enough for transplant treatment (kidney, lung, heart, liver, and blood clotting limits)
- You can commit to study visits and monitoring, and (if you could become pregnant) you agree to effective birth control during treatment and recovery
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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