One-Shot gene therapy trial offers new hope for gaucher disease
NCT ID NCT06818838
Summary
This early-stage study is testing a new, one-time gene therapy called LY-M001 for adults with Gaucher Disease Type 1. The goal is to see if a single injection is safe and can help the liver produce a crucial missing enzyme for a long time, which could reduce harmful substance buildup in the body. The study will first test different doses in a small group of up to 12 participants to find the safest and most effective amount.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Guangzhou First People's Hospital
RECRUITINGGuangzhou, Guangdong, 510000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Hematology Hospital, Chinese Academy of Medical Sciences
RECRUITINGTianjin, Tianjin Municipality, 300011, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Shanxi Bethune Hospital
RECRUITINGTaiyuan, Shanxi, 030000, China
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Conditions
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