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Experimental drug ION717 aims to slow fatal brain disease

NCT ID NCT06153966

Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 18, 2026 · Updated 9 times

Summary

This study tests a new drug called ION717 for people with early-stage prion disease, a rare and fatal brain condition. The drug is given via a spinal injection and aims to reduce harmful prion proteins. The trial will check safety, side effects, and how the drug moves through the body. About 76 participants with a caregiver are needed.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

85 people

The number who actually took part.

Started

Jan 2024

Expected to finish

Aug 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

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 * A confirmed diagnosis of probable or definite prion disease. * Early-stage prion disease at the time of Screening. * Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits. * Patients must have a caregiver who is ≥ 18 years old and who is able and willing to facilitate the patient's involvement, to the best of their ability, for the duration of the trial; caregivers must also be able and willing to provide information about themselves and the patient for the duration of the trial. * Aged ≥ 18 at the time of informed consent. Key Exclusion Criteria * Clinically significant abnormalities in medical history, laboratory tests or physical examination that would render a patient unsuitable for inclusion. * Any contraindication or unwillingness to undergo an MRI. * Obstructive hydrocephalus, presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter. * Known brain or spinal disease that would interfere with the LP process, CSF circulation or safety assessment. * Have any other condition, which, in the opinion of the Investigator would make the patient unsuitable for inclusion or could interfere with the patient participating in or completing the study.

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Conditions

The condition(s) this trial relates to.

prion disease

As listed by the trial registrant

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

Prion disease

Contacts and locations

Locations

  • Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Hospital Clinic De Barcelona

    Barcelona, 08036, Spain

  • Hôpital Universitaire Pitié Salpêtrière

    Paris, 75013, France

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • McGill University Health Centre

    Montreal, Quebec, H3A 2B4, Canada

  • Mihara Memorial Hospital

    Gunma, Isesaki-shi, 372-0006, Japan

  • NYU Langone Health

    New York, New York, 10016, United States

  • National Center of Neurology and Psychiatry

    Tokyo, Kodaira-shi, 187-8551, Japan

  • Neuromuscular Center Yoshimizu Hospital

    Yamaguchi, Shimonoseki-shi, 751-0826, Japan

  • Royal Melbourne Hospital

    Parkville, 3050, Australia

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 6423906, Israel

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • University Medical Center Göttingen

    Göttingen, 37075, Germany

More trials for these conditions

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