Plain-English translation of NCT06379789 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Hemophilia B research guide →Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing -, a new gene therapy designed to help people with hemophilia B. The medication uses a technology called CRISPR to insert instructions into your liver cells so your body can make its own clotting protein (called Factor IX). The goal is to help reduce or stop bleeding episodes and reduce dependence on regular clotting factor infusions.
People with hemophilia B have difficulty making a clotting protein their bodies need to stop bleeding. Currently, patients must receive regular infusions of this clotting factor for their entire lives. This medication offers the possibility of a one-time treatment that could give the body the ability to make its own clotting protein, potentially changing how hemophilia B is managed.
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If you join this study, you would first complete a 6-month lead-in period where researchers track your bleeding episodes while you continue your regular clotting factor infusions. Then you would receive a single dose of the medication through an IV infusion. After that, you would have regular follow-up visits — more frequent at first, then spaced out over time — where doctors check your blood clotting levels, monitor for side effects, and track how often you bleed. The study is designed in stages, starting with adults to check safety before enrolling teens and children.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
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