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Scientists to examine liver tissue after gene therapy for hemophilia

NCT ID NCT05932914

Knowledge-focused Sponsor: St. Jude Children's Research Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Sep 18, 2026 · Updated 7 times

Summary

This observational study will take small liver samples from 8 adults with hemophilia A or B who had gene therapy at least 6 months ago. Researchers want to see how the gene therapy affected liver cells, including how many cells still carry the therapy and whether there is any liver damage. The goal is to learn more about the long-term safety and effects of gene therapy on the liver.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study could help researchers understand how gene therapy affects the liver over time, potentially improving future treatments for hemophilia.
What could go wrong
This is a very small observational study (8 people) that does not test a new treatment. It only looks at past gene therapy recipients, so results may not apply to everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 8 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Oct 2026

An estimate. Start dates often move.

Expected to finish

Jan 2030

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Those who meet the Eligibility Criteria.

Ages

18 to 80 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 (Do not apply to participants who will not undergo liver biopsy, and have leftover liver tissue from a previous biopsy procedure, because all exclusion criteria only cover the safety considerations for the biopsy procedure.): * 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

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Conditions

The condition(s) this trial relates to.

hemophilia A hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia A Hemophilia B

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

    Contact Email: •••••@•••••

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