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Liver Biopsy Following Gene Therapy For Hemophilia

NCT ID: NCT05932914

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

8 participants

Study Classification

OBSERVATIONAL

Study Start Date

2026-01-31

Study Completion Date

2030-01-31

Brief Summary

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This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have previously been treated with a factor VIII or factor IX gene-containing AAV-vector for liver-targeted gene transfer. Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion.

Detailed Description

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This is a prospective cohort study evaluating the effect of AAV-mediated gene therapy on the liver in adult patients with hemophilia A or hemophilia B who have previously been treated with a FVIII/FIX gene-containing AAV-vector for liver-targeted gene transfer.

To better understand the effect of AAV-mediated gene transfer on the liver, eligible participants can allow the use and analysis of already existing liver tissue samples, taken and preserved after having received gene therapy. Alternatively, eligible participants can consent to providing a new liver tissue sample which will be obtained by undergoing a standard transjugular liver biopsy (TJLB) procedure under moderate sedation. . The procedure will be done as outpatient (day 1) with post procedure follow-up on day 2, 3, 4 and 14.

Conditions

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Hemophilia A Hemophilia B

Keywords

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AAV-mediated factor VIII (FVIII) gene transfer AAV-mediated factor IX (FIX) gene transfer Gene Therapy Hemophilia A Hemophilia B Transjugular liver biopsy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Participants

Those who meet the Eligibility Criteria

Liver Biopsy

Intervention Type PROCEDURE

Standard transjugular liver biopsy under moderate sedation

Interventions

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Liver Biopsy

Standard transjugular liver biopsy under moderate sedation

Intervention Type PROCEDURE

Eligibility Criteria

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Inclusion Criteria

* 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 Criteria

* 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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Methodist University Hospital

UNKNOWN

Sponsor Role collaborator

St. Jude Children's Research Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Ulrike Reiss, MD

Role: PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital

Locations

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St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

Countries

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United States

Central Contacts

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Ulrike Reiss, MD

Role: CONTACT

Phone: 866-278-5833

Email: [email protected]

Facility Contacts

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Ulrike Reiss, MD

Role: primary

Related Links

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http://www.stjude.org

St. Jude Children's Research Hospital

http://www.stjude.org/protocols

Clinical Trials Open at St. Jude

Other Identifiers

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LIVBX

Identifier Type: -

Identifier Source: org_study_id