Scientists to examine liver tissue after gene therapy for hemophilia

NCT ID NCT05932914

Not yet recruiting Knowledge-focused Sponsor: St. Jude Children's Research Hospital Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 5 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.

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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

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Contact Email: •••••@•••••

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