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Liver Biopsy After Gene Therapy for Hemophilia

This study is looking at the long-term effects of gene therapy on the liver in adults with hemophilia A or B. Participants previously received gene therapy using an AAV vector (a type of virus used to deliver genes) containing either the factor VIII or factor IX gene. Researchers will examine liver tissue samples to see how many liver cells have taken up the AAV vector. You must be between 18 and 80 years old, male, and have participated in specific past gene therapy trials (AGT4HB or GO8). This is an observational study, meaning researchers are looking at what has already happened or is happening, rather than testing a new treatment. The study aims to enroll 8 participants, but its current status is unclear.

Study design
This is an observational study with a planned enrollment of 8 male participants, aged 18-80. It is a prospective cohort study, meaning participants are followed forward in time.
What's involved
You would either allow researchers to use existing liver tissue samples or undergo a standard transjugular liver biopsy (a procedure to take a small piece of liver tissue) under moderate sedation. This procedure is done as an outpatient, with follow-up visits on days 2, 3, 4, and 14.
Compensation
Not stated in the trial record.
Follow-up
After the liver biopsy procedure, there are follow-up visits on days 2, 3, 4, and 14.

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NCT05932914

Liver Biopsy Following Gene Therapy For Hemophilia

Not Yet Recruiting
Not specifiedAges 18–80Observational
St. Jude Children's Research Hospital
~8 participants
Updated 2026-08-13 on ClinicalTrials.gov
What's tested:Liver Biopsy

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Assessment of the number of hepatocytes transduced with AAV vector genome in liver biopsy samples analyzed by FISH
Measured over single time point (day of biopsy)

Conditions

Hemophilia A
Hemophilia B

Explore related trials

Hemophilia AHemophilia B

Where it's being run

1 sites across 1 states
Tennessee1

Study leadership

  • Ulrike Reiss, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital

Who to contact

Ulrike Reiss, MD
[email protected]
888-226-4343
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Age ≥18 to 80 years
Patients, who were enrolled and treated in one of the following clinical trials:
AGT4HB (EudraCT number: 2005-005711-17; NCT00979238) - FIX AAV gene therapy trial (sponsor: St. Jude Children's Research Hospital)
GO8 (EudraCT number:2014-003880-38; NCT02576795) - FVIII AAV gene therapy trial (sponsor: University College, London)
Able to give informed consent
Able to comply with study requirements

Exclusion

Any condition that, in the opinion of the investigator or sponsor of the ongoing clinical trial in which the patient is participating in, would prevent the patient from fully complying with the requirements of the clinical trial and/or would influence or interfere with evaluation and interpretation of subject safety or efficacy result of that ongoing clinical trial
Platelet count \<140x10\^9/L
INR \>1.5
Abnormal kidney function with estimated GFR \<50 mL/min (calculated using the CKD-EPI equation)
Known allergy to iodine-based intravenous contrast agents
Known allergy to local or general anesthetics
Known allergic reaction to FVIII/FIX concentrate infusions
Presence of FVIII inhibitor or FIX inhibitor (historical result can be used if done within 14 weeks of this liver biopsy)
Evidence of any bleeding disorder other than hemophilia A or B

What this trial measures

  • Assessment of the number of hepatocytes transduced with AAV vector genome in liver biopsy samples analyzed by FISHsingle time point (day of biopsy)

    Fluorescence in situ hybridization (FISH)