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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This trial tests giving a stem cell transplant to a fetus with Fanconi anemia before birth, using the mother's bone marrow cells. The goal is to help the baby's body make healthy blood cells before symptoms start.
This Phase 2 study tests a stem cell transplant approach using adjusted chemotherapy doses for people with Fanconi anemia. It may help replace damaged bone marrow and treat blood problems like severe aplastic anemia, MDS, or leukemia.
This Phase 2 study tests a special “T-cell depleted” transplant plan for people with inherited bone marrow failure disorders (Fanconi anemia or T-Beta-thalassemia—or “TBD”). It may help restore healthy blood production while lowering the chance of certain transplant complications.
This Phase 2 trial tests a stem cell (bone marrow) transplant approach to restore bone marrow function and immune function in people with certain inherited immune problems. If you have one of these conditions and a suitable donor, the study may offer a path to rebuild blood and immune systems.
This trial tests whether a gentler (reduced-intensity) conditioning treatment before a stem cell transplant works for non-cancer diseases. It uses specific donor stem cell matches and checks heart, lung, liver, and kidney function to help lower transplant stress while still aiming for benefit.
This study follows people with Fanconi anemia (a bone marrow failure condition) who receive a transplant from a half-matched (haploidentical) donor. It aims to better understand outcomes and care needs for patients with severe blood counts and higher-risk disease.
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