Plain-English translation of NCT04817462 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
This study doesn't follow the usual testing phases — it may be an observational study or a different type of research.
Researchers want to better understand how AAV gene therapy—a treatment that uses a modified virus to deliver working genes into liver cells—affects your liver over time. By studying liver tissue samples from people who have already received this treatment, they can learn whether it's working safely and effectively, and use that knowledge to help future patients.
Gene therapy for hemophilia is promising, but doctors still have questions about exactly how it works in the human body and what happens to your liver cells over the long term. This study fills that gap by examining actual tissue samples from patients who have already benefited from the treatment.
You likely qualify if…
You likely don't qualify if…
You will undergo a liver biopsy, a procedure where doctors use imaging guidance to safely collect a small sample of liver tissue. This is typically done as a single outpatient visit. The tissue sample will then be analyzed in the laboratory to help researchers understand how your liver cells are responding to the gene therapy treatment.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
United Kingdom
Sponsor
University College, London
Enrollment target
~10 participants
Started
August 2022
Primary completion
July 2025
This trial's estimated completion date has passed — the record may not be fully up to date.
Age range
18 Years – 80 Years
Sex
Male only
Last updated on clinicaltrials.gov in December 2024.
Reach out to the team running this trial. Response times vary — some teams are faster than others.
Central contact
Paul Batty
Royal Free Hospital NHS Foundation Trust
Tell us you're interested and we'll help connect you with the research team. We'll walk you through what to expect first — no email needed to get started.