One-Shot gene therapy trial offers hope for gaucher disease patients
NCT ID NCT05487599
Summary
This early-stage study is testing a single intravenous dose of an experimental gene therapy called LY3884961 in adults with Gaucher disease. The main goal is to see if the treatment is safe and tolerable, while also checking if it can improve key disease measures like spleen size and blood counts. Up to 15 participants will be followed for about 5 years to monitor long-term effects.
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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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