One-Shot gene fix tested in kids with rare, dangerous fat disorder
NCT ID NCT07371767
Summary
This early-stage study is testing a new, one-time gene editing treatment called CS-121 in children and teenagers with a rare, severe genetic disorder that causes extremely high blood fats. The goal is to see if the treatment is safe and can lower fat levels by editing a specific gene in the liver, which could reduce the risk of life-threatening pancreatitis. The study will enroll up to 15 young patients to find the best dose.
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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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Shanghai Children's Medical Center
RECRUITINGShanghai, Shanghai Municipality, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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