Plain-English translation of NCT05709288 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Hemophilia B research guide →Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This is an early-stage trial testing a new treatment called , a gene therapy designed to help teenagers with hemophilia B. The medication works by delivering genetic instructions through a modified virus that helps the body produce more of its own clotting factor (factor IX). If successful, this treatment could reduce or eliminate the need for regular infusions.
Teenagers with severe hemophilia B currently require frequent infusions of clotting factor to prevent dangerous bleeding. This medication is being tested to see if a one-time gene therapy approach could offer a longer-lasting solution by helping the body make its own clotting factor.
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You would receive a single intravenous infusion of the medication at the hospital or clinic. After that, you would return for safety check-ups over the next year, with additional follow-up visits continuing for up to 10 years to monitor how well the treatment works and ensure it remains safe. Blood tests and physical exams will be part of your regular visits.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
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