First human trial of RNA therapy for fatal brain disease begins
NCT ID NCT07444580
First seen Mar 09, 2026 · Last updated May 15, 2026 · Updated 7 times
Summary
This early-stage trial tests a new drug called PrP-siRNA in 30 adults with symptomatic prion disease, a rare and fatal brain condition. The drug is given via a spinal injection and aims to reduce levels of the harmful prion protein. The main goal is to check safety, but researchers will also measure how well the drug lowers prion protein in the spinal fluid.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Columbia University Medical Center
RECRUITINGNew York, New York, 10032, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Contact
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Massachusetts General Hospital
RECRUITINGBoston, Massachusetts, 02114, United States
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Mayo Clinic
RECRUITINGRochester, Minnesota, 55905, United States
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Vanderbilt University Medical Center
RECRUITINGNashville, Tennessee, 37232, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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