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 type of gene therapy for people with hemophilia B, a serious bleeding disorder. The medication uses CRISPR technology—a tool that edits genes—to help your body produce the clotting factor (Factor IX) that people with hemophilia B don't make enough of. The goal is to see if this one-time treatment can reduce or eliminate the need for regular factor injections.
People with hemophilia B currently need frequent injections of clotting factor to prevent dangerous bleeding. This medication aims to offer a longer-lasting solution by fixing the underlying genetic problem, so your body can make its own clotting factor instead of relying on injections.
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The study has two main phases. In Part 1, adult participants receive gradually increasing doses of the medication to find the safest and most effective dose. In Part 2, more participants (adults, teenagers, and children as young as 2 years old) receive the dose determined in Part 1. You would receive one infusion of the medication, then return for regular visits over time—including blood tests and assessments—to monitor how well the treatment works and check for any side effects.
AI-generated summary from trial data · Jul 14, 2026 · Not medical advice
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