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Experimental spinal injection aims to slow rare brain disease

NCT ID NCT04165486

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests an experimental drug called ION464 in 40 adults with multiple system atrophy (MSA), a rare and progressive brain disorder. The drug is given via a spinal injection and aims to reduce levels of a protein linked to the disease. The main goal is to check safety and tolerability, not yet to prove it works.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ION464 (an experimental drug given via spinal injection)
What this could lead to
If successful, this could point toward a treatment that slows or stops the progression of multiple system atrophy, a rare and devastating brain disease.
What could go wrong
This is a very early Phase 1 trial with only 40 participants, focused on safety. The drug may not work, could have side effects, and it will take years to know if it truly helps patients.

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 40 people

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

Started

Jul 2022

Expected to finish

Sep 2027

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

40 to 70 years

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: * Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with qualitative, visual read. * Diagnosed with probable or possible MSA, either parkinsonian-type (MSA-P) or cerebellar-type (MSA-C). * Must be able to walk unassisted for at least 10 meters (approximately 30 feet) Key Exclusion Criteria: * Presence of cognitive dysfunction (defined as Montreal Cognitive Assessment (MoCA) score \<25) * Family history of ataxia or parkinsonism and known genetic cause of ataxia or parkinsonism. NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

multiple system atrophy

As listed by the trial registrant

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

Multiple system atrophy

How to take part

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

  1. The places running it

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

  2. 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 ↗

  3. 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

Locations

  • CHU de Lyon - Hospices Civils de Lyon-H6pital Pierre Wertheimer, Neurologique HCL

    RECRUITING

    Lyon, 69500, France

  • Groupe Hospitalier Pitie-Salpetriere

    RECRUITING

    Paris, 75013, France

  • Hopital Purpan

    RECRUITING

    Toulouse, 31059, France

  • Hospital Beatriz Ângelo

    RECRUITING

    Loures, 2674-514, Portugal

  • Institute of Neurology & The National Hospital for Neurology and Neurosurgery

    RECRUITING

    London, England, WC1N 3BG, United Kingdom

  • Klinikum der Universtiatet Muenchen -Campus Grosshadern

    RECRUITING

    München, 81377, Germany

  • Medizinische Hochschule Hannover (MHH)

    COMPLETED

    Hanover, 30625, Germany

  • Medizinische Universität Innsbruck

    RECRUITING

    Innsbruck, 6020, Austria

  • Salford Royal Hospital

    RECRUITING

    Salford, M6 8HD, United Kingdom

  • Southampton General Hospital

    RECRUITING

    Southampton, SO16 6YD, United Kingdom

  • The John Radcliffe Hospital

    RECRUITING

    Oxford, England, OX3 9DU, United Kingdom

  • Universitaetsklinikum Duesseldorf AoeR

    RECRUITING

    Düsseldorf, 40225, Germany

  • Universitaetsklinikum Ulm

    RECRUITING

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University Hospital Marburg

    RECRUITING

    Marburg, 35043, Germany

  • University Medical Center Göttingen, Clinic for Neurology

    RECRUITING

    Göttingen, 37075, Germany

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