MedTrace Logo

Liver Biopsy In Haemophilia Gene Therapy

NCT04817462 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2024-12-20

No results posted yet for this study

Summary

To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX/FVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.

Conditions

  • Hemophilia B, Severe
  • Hemophilia A, Severe

Interventions

PROCEDURE

Liver biopsy

The study population is patients with either haemophilia A or B who have previously been administered gene therapy treatment in one of three specific gene therapy clinical trials. In this study they will have a liver biopsy performed to take up to 3 samples for laboratory analysis.

Sponsors & Collaborators

  • University College, London

    lead OTHER

Principal Investigators

  • Paul Batty, MBBS MRCP · Royal Free Hospital NHS Foundation Trust

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-08-05
Primary Completion
2025-07-01
Completion
2025-07-01

Countries

  • United Kingdom

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04817462 on ClinicalTrials.gov