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One-Time gene shot could free gaucher patients from lifelong infusions

NCT ID NCT05487599

Recruiting now Disease control Sponsor: Prevail Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 14, 2026 · Updated 2 times

Summary

This early-stage study tests a single intravenous dose of a gene therapy called LY3884961 in 15 adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or replace the need for ongoing enzyme replacement or substrate reduction therapy. Participants must have been on stable standard treatment for at least 2 years. The study lasts about 5 years, with close monitoring for side effects and disease markers.

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Conditions

The condition(s) this trial relates to.

Gaucher disease Gaucher disease type I

As listed by the trial registrant

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

GAUCHER DISEASE GAUCHER DISEASE, TYPE 1

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Ann and Robert H Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Cedars-Sinai

    RECRUITING

    Los Angeles, California, 90048, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Duke University Health System

    RECRUITING

    Durham, North Carolina, 27710-3017, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn

    RECRUITING

    Zaragoza, 50006, Spain

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Hospital de Clinicas de Porto Alegre (HCPA)

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Lysosomal & Rare Disorders Research and Treatment Center

    RECRUITING

    Fairfax, Virginia, 22030-6066, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Royal Free Hospital NHS Trust

    RECRUITING

    London, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • SphinCS Clinical Science for LSD

    RECRUITING

    Höchheim, 65239, Germany

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Westmead Hospital-Cnr Hawkesbury and Darcy Rds

    COMPLETED

    Westmead, New South Wales, 2145, Australia

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