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Could a single injection silence fatal brain disease?

NCT ID NCT07444580

Disease control Sponsor: Broad Institute of MIT and Harvard Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 1 time

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key inclusion criteria: 1. clinically manifested symptoms of prion disease, in the opinion of the investigator; 2. a diagnosis of probable prion disease according to CDC criteria; 3. a positive CSF RT-QuIC or PRNP genetic test; 4. no more than moderate functional impairment as quantified by an MRC-PDRS score ≥15; and 5. availability of a study partner to assist with study procedures. Key exclusion criteria: 1. pregnancy; 2. contraindication to LP; or 3. recent participation in a different prion disease clinical trial. Additional inclusion and exclusion criteria apply and will be evaluated at screening.

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Conditions

The condition(s) this trial relates to.

Creutzfeldt Jacob disease fatal familial insomnia Gerstmann-Straussler-Scheinker Disease prion disease

As listed by the trial registrant

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

Prion disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    5 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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

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