Could a single shot replace daily gaucher meds?
NCT ID NCT05487599
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 30 times
Summary
This study tests a one-time gene therapy (LY3884961) for adults with Gaucher disease type 1. The goal is to see if it is safe and can reduce or stop the need for current enzyme replacement or substrate reduction therapy. About 15 participants will receive a single intravenous dose and be followed for 5 years.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann and Robert H Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Duke University Health System
RECRUITINGDurham, North Carolina, 27710-3017, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Quironsalud Zaragoza, Paseo Mariano Renovales Sn
RECRUITINGZaragoza, 50006, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital de Clinicas de Porto Alegre (HCPA)
RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-903, Brazil
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Lysosomal & Rare Disorders Research and Treatment Center
RECRUITINGFairfax, Virginia, 22030-6066, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Free Hospital NHS Trust
RECRUITINGLondon, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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SphinCS Clinical Science for LSD
RECRUITINGHöchheim, 65239, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Westmead Hospital-Cnr Hawkesbury and Darcy Rds
COMPLETEDWestmead, New South Wales, 2145, Australia
Conditions
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