Study drug for adults with hemophilia B who still bleed
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1/2 study tests ZS801, a gene-related treatment, to see how safe it is and whether it helps prevent bleeding in adults with hemophilia B. You may be considered if you’ve had frequent bleeds or joint problems and meet several lab and health requirements.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are a man age 18 or older
- You have confirmed hemophilia B with low natural clotting factor (FIX) at 2% or less
- You have had at least 100 days of prior FIX treatment (recorded exposure days) and continued symptoms (at least 3 bleeding events and/or joint arthritis needing FIX in the last year)
- You must be able and willing to use reliable birth-control methods during the study and for 52 weeks after the ZS801 dose, and you must not donate sperm during that time
- You must be able to take related medicines if needed (like steroid or immune-suppressing drugs) and tolerate them
- You must not have a FIX “inhibitor” (an immune reaction that blocks FIX), and you must not have certain serious illnesses or abnormal blood tests
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 studies ZS801, a gene therapy, in adults with hemophilia B who have very low natural FIX (a clotting protein) and frequent bleeding or joint problems. It aims to see how well it works and whether it is safe, especially by monitoring blood counts, infections, and immune responses.
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This trial tests a one-time (or limited) gene therapy meant to help the body make more factor IX (FIX), which can reduce bleeding in people with hemophilia B. You may be able to qualify if you have significant bleeding history, no factor IX “blockers” (inhibitors), and can follow strict safety and pregnancy-prevention rules.
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