Liver Biopsy Following Gene Therapy For Hemophilia

Part of paid clinical trials in Memphis, Tennessee.

Sponsor
St. Jude Children's Research Hospital
Study ID
NCT05932914
Status
Not Yet Recruiting

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Conditions

  • Hemophilia A
  • Hemophilia B

Eligibility Criteria

Sex
MALE
Age
18 Years - 80 Years
Healthy Volunteers
Not accepted

Interventions

  • Liver Biopsy — PROCEDURE
    Standard transjugular liver biopsy under moderate sedation

Study Details

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.

Key Dates

Start date
Jul 31, 2026
Status verified
Jun 2026
Primary completion
Jan 31, 2030
Completion
Jan 31, 2030

Study Design

Enrollment
8 participants (estimated)

Arms

  • Arm: Participants
    Those who meet the Eligibility Criteria

Primary Outcome Measure

Assessment of the number of hepatocytes transduced with AAV vector genome in liver biopsy samples analyzed by FISH [ Time Frame: single time point (day of biopsy) ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
St. Jude Children's Research HospitalMemphisTennessee38105
Ulrike Reiss, MD
[email protected]
888-226-4343

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By research site
St. Jude Children's Research Hospital· Memphis, TN

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