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New Huntington's drug shows promise in Mid-Stage trial

NCT ID NCT05358717

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested a drug called PTC518 in 159 adults with Huntington's disease to see if it is safe and can lower a harmful protein in the blood. Participants received different doses of the drug or a placebo for up to 12 months. The goal is to slow disease progression by targeting the root cause.

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

159 people

The number who actually took part.

Started

Jun 2022

Finished

Jul 2025

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.

Key Inclusion Criteria: * Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length from 40 to 50, inclusive Eligibility for HD-ISS Stage 2 Group (Parts A, B, and C): * A Unified Huntington's Disease Rating Scale (UHDRS)-Independence Scale (IS) score of 100 * A UHDRS Total Functional Capacity (TFC) score of 13 * A score between 0.18 and 4.93 inclusive on the normed version of the HD prognostic index (PINHD) Eligibility for HD-ISS Mild Stage 3 Group (Parts D, E, and F): * A UHDRS Total Functional Capacity (TFC) score of 11 or 12, or a UHDRS TFC score of 13 with an UHDRS IS score of \<100 Key Exclusion Criteria: * Receipt of an experimental agent within 90 days or 5 half-lives prior to Screening or anytime over the duration of this study, ribonucleic acid (RNA)- or deoxyribonucleic acid (DNA)-targeted HD-specific investigational agents such as antisense oligonucleotides, cell transplantation, or any other experimental brain surgery * Any history of gene therapy exposure for the treatment of HD * Participation in an investigational study or investigational paradigm (such as exercise/physical activity, cognitive therapy, brain stimulation, etc) within 90 days prior to Screening or anytime over the duration of this study * Any medical history of brain or spinal disease that would interfere with the lumbar puncture process safety assessments * Any medical history or condition that would interfere with the ability to complete the protocol-specified assessments (for example, implanted shunt, conditions precluding magnetic resonance imaging \[MRI\] scans) * Pregnancy, planning on becoming pregnant during the course of the study or within 6 months of end of treatment, or currently breastfeeding 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

  • Brain and Spine Institute Paris

    Paris, 75013, France

  • Cardiff University Schools of Medicine and Biosciences

    Cardiff, CF10 3AX, United Kingdom

  • Centre Hospitalier Universitaire d'Angers

    Angers, 49100, France

  • Charite University Medicine Berlin

    Berlin, 10117, Germany

  • George-Huntington-Institut

    Münster, 48149, Germany

  • Hospital Ramón y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario Burgos

    Burgos, 90550, Spain

  • Hospital Universitario Cruces

    Barakaldo, 48903, Spain

  • Hospital de la Santa Creu I Sant Pau

    Barcelona, 8025, Spain

  • Hôpital Universitaire de Marseille Hôpital de la Timone

    Marseille, 13385, France

  • IRCCS Carlo Besta Neurological Institutte

    Milan, 20133, Italy

  • IRCCS Casa Sollievo della Sofferenza Research Hospital

    San Giovanni Rotondo, 71013, Italy

  • Irccs Istituto Delle Scienze Neurologiche Di Bologna Uoc Clinica Neurologica

    Bologna, 40139, Italy

  • Leiden University Medical Center

    Leiden, 2333 ZA, Netherlands

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL, United Kingdom

  • Medical University Innsbruck

    Innsbruck, 6020, Austria

  • Monash Health

    Clayton, 3168, Australia

  • Ruhr-Univ. Bochum St. Joseph-Hospital

    Bochum, 44791, Germany

  • The Barbery National Centre for Mental Health

    Birmingham, B15 2FG, United Kingdom

  • The Ottawa Hospital, Parkinson's and Movement Disorders Clinic

    Ottawa, K1Y 4E9, Canada

  • The University of Texas Health Science Center at Houston; McGovern Medical School

    Houston, Texas, 77030, United States

  • UCL Queen Square Institute of Neurology National Hospital for Neurology and Neurosurgery

    London, WC1N 3BG, United Kingdom

  • Ulm University, UKU, Dep. of Neurology

    Ulm, 89081, Germany

  • University of California, San Diego

    La Jolla, California, 92037, United States

  • University of Otago, New Zealand Brain Research Institute

    Christchurch, 8011, New Zealand

  • University of Washington Department of Neurology

    Seattle, Washington, 98195, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37212, United States

  • Westmead Hospital

    Sydney, 2145, Australia

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