Plain-English translation of NCT06003387 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Hemophilia B research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial is testing CSL222, a one-time gene therapy designed to help adults with severe hemophilia B — a serious bleeding disorder. Most people with hemophilia B need regular infusions of a clotting factor to prevent dangerous bleeding. This medication uses a modified virus to deliver a working copy of a gene directly into your body, which could reduce or eliminate your need for those regular infusions.
Many people with hemophilia B have developed antibodies against the virus used in gene therapy, which can prevent the treatment from working properly. This trial exists to see whether this medication can still be safe and effective for people in this situation who have fewer options.
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You would receive a single intravenous infusion of the medication on Day 1 of the study. The research team would monitor you closely over time with clinic visits and blood tests to see how well the treatment works and how safe it is. You would also keep a diary of any bleeding episodes or symptoms, and continue working with your care team throughout the study to track your progress.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
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