Clin2
NCT04817462Possibly a fitRecruiting

Liver biopsy in people with haemophilia gene therapy

Hemophilia B, SevereHemophilia A, Severe

Part of Blood & lymphatic, Genetic & congenital clinical trials.

This study takes a small liver tissue sample (a liver biopsy) in men who previously received haemophilia gene therapy. The goal is to learn more about how the gene therapy works and how safe it is for the liver.

Summary written for real people, not researchers, by Clin2.

Phase
N/A
Enrollment
10 people
Ages
18 years to 80 years
Study type
Interventional

Who can take part

  • You are a man age 18 to 80
  • You previously joined and were treated in one of these gene therapy trials at Royal Free Hospital: AGT4HB, GO-8, or FLT180a-01 (or follow-up FLT180a-04)
  • Your blood clotting protein level (FVIII for haemophilia A or FIX for haemophilia B) is above 1% at some point after gene therapy, with normal PT and TT blood tests
  • Your platelet count is at least 140 (not low)
  • Your kidney function is normal enough (GFR 50 or higher)
  • You can safely have the biopsy: no iodine contrast allergy, no anaesthetic allergy, no known problems reacting to FVIII/FIX infusions, and no FVIII/FIX inhibitor present within the last 14 weeks

View the official record on ClinicalTrials.gov

Quick eligibility check

Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.

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