Gene therapy study for adults with hemophilia A
Part of Blood & lymphatic, Genetic & congenital clinical trials.
This Phase 1/2 study tests a one-time gene therapy (ASC618) to help the body make clotting factor for people with hemophilia A. It may reduce bleeding and the need for factor infusions, but you must meet several safety and eligibility checks.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are male and at least 18 years old.
- You have hemophilia A that is severe or moderately severe (very low factor VIII activity).
- If you use factor only when you bleed, you had at least 12 bleeding episodes in the last 12 months; if you use regular prevention, you’ve had at least 150 days of exposure to factor VIII treatment.
- Your body mass index (BMI) is 30 or less.
- You do not have certain gene-therapy “background immunity”: no prior AAV8 antibodies or neutralizing antibodies on required blood tests.
- You can meet pregnancy-prevention rules after the infusion (double-barrier contraception until semen tests are negative).
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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