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

NCT ID NCT05358717

Completed Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

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.

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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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