Could a single injection silence fatal brain disease?

NCT ID NCT07444580

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests an experimental 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 the harmful prion protein in the brain. The main goal is to check safety and find the right dose, while also measuring how well it lowers the protein levels.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Columbia University Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • University Hospitals Cleveland Medical Center

    RECRUITING

    Cleveland, Ohio, 44106, United States

  • Vanderbilt University Medical Center

    RECRUITING

    Nashville, Tennessee, 37232, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.