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New hope for Huntington's: Long-Term drug trial underway

NCT ID NCT06254482

Disease control Sponsor: Novartis Pharmaceuticals 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This study looks at the long-term safety and effects of the drug votoplam in people with Huntington's disease. About 144 participants who finished a previous votoplam study will take part. The goal is to see how the drug affects Huntington's protein levels and brain changes over time.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

144 people

The number who actually took part.

Started

Aug 2023

Expected to finish

Jul 2029

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

25 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.

Inclusion Criteria: * Participant who has completed the Treatment Period in Study PTC518-CNS-002-HD. Exclusion Criteria: * Participants who have not previously completed the Treatment Period in Study PTC518-CNS-002-HD. Note: Other inclusion and exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

Huntington disease neurodegenerative disease Rare Diseases

As listed by the trial registrant

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

Huntington disease

Contacts and locations

Locations

  • Novartis Investigative Site

    Clayton, 3168, Australia

  • Novartis Investigative Site

    Westmead, 2145, Australia

  • Novartis Investigative Site

    Innsbruck, 6020, Austria

  • Novartis Investigative Site

    Ottawa, K1Y 4E9, Canada

  • Novartis Investigative Site

    Angers, 49100, France

  • Novartis Investigative Site

    Marseille, 13385, France

  • Novartis Investigative Site

    Paris, 75013, France

  • Novartis Investigative Site

    Berlin, 10117, Germany

  • Novartis Investigative Site

    Bochum, 44791, Germany

  • Novartis Investigative Site

    Münster, 48149, Germany

  • Novartis Investigative Site

    Ulm, 89081, Germany

  • Novartis Investigative Site

    Bologna, 40139, Italy

  • Novartis Investigative Site

    Milan, 20133, Italy

  • Novartis Investigative Site

    San Giovanni Rotondo, 71013, Italy

  • Novartis Investigative Site

    Leiden, 2333 ZA, Netherlands

  • Novartis Investigative Site

    Christchurch, 8011, New Zealand

  • Novartis Investigative Site

    Barakaldo, 48903, Spain

  • Novartis Investigative Site

    Barcelona, 8025, Spain

  • Novartis Investigative Site

    Burgos, 90550, Spain

  • Novartis Investigative Site

    Madrid, 28034, Spain

  • Novartis Investigative Site

    Birmingham, B15 2FG, United Kingdom

  • Novartis Investigative Site

    Cardiff, CF14 4XW, United Kingdom

  • Novartis Investigative Site

    London, WC1N 3BG, United Kingdom

  • Novartis Investigative Site

    Manchester, M13 9WL, United Kingdom

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