An Extension Study to Evaluate the Long-Term Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)

Last updated: July 9, 2025
Sponsor: PTC Therapeutics
Overall Status: Active - Enrolling

Phase

2

Condition

Dyskinesias

Treatment

PTC518

Clinical Study ID

NCT06254482
PTC518-CNS-004-HD
2023-504628-24-00
  • Ages > 25
  • All Genders

Study Summary

The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of PTC518 in participants with HD.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Participant who has completed the Treatment Period in Study PTC518-CNS-002-HD.

Exclusion

Exclusion Criteria:

  • Participants who have not previously completed the Treatment Period in StudyPTC518-CNS-002-HD.

Note: Other inclusion and exclusion criteria may apply.

Study Design

Total Participants: 250
Treatment Group(s): 1
Primary Treatment: PTC518
Phase: 2
Study Start date:
August 25, 2023
Estimated Completion Date:
July 20, 2027

Study Description

Participants who completed the Treatment Period in the parent Phase 2a Study PTC518-CNS-002-HD (NCT05358717), fulfilled the enrollment criteria, and chose to enroll in this extension study will undergo baseline evaluations and be assessed for 54 additional months.

All participants will receive active PTC518 in this extension study. Participants who received PTC518 in the parent Study PTC518-CNS-002-HD will continue at the same dose level they received in that study in a blinded fashion (5, 10, or 20 milligrams [mg]). Participants who received placebo in the parent Study PTC518-CNS-002-HD will be allocated to a PTC518 dose level according to the same dosing group in which they were previously randomized (5, 10, or 20 mg). The 20 mg dosing arm will be open for randomization only if and when the Data Safety and Monitoring Board from the parent Study PTC518-CNS-002-HD recommends initiating this dosing level based on an unblinded review of safety data from that study.

Connect with a study center

  • Monash Health

    Clayton, 3168
    Australia

    Site Not Available

  • Westmead Hospital

    Westmead, 2145
    Australia

    Site Not Available

  • Medical University Innsbruck

    Innsbruck, 6020
    Austria

    Site Not Available

  • Centre hospitalier de l'université de Montréal (CHUM)

    Montreal, QC H2X 3H8
    Canada

    Site Not Available

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

    Ottawa, K1Y 4E9
    Canada

    Site Not Available

  • University of British Columbia (UBC)

    Vancouver, V6T 2B5
    Canada

    Site Not Available

  • Centre Hospitalier Universitaire d'Angers

    Angers, 49100
    France

    Site Not Available

  • Hôpital Universitaire de Marseille Hôpital de la Timone

    Marseille, 13385
    France

    Site Not Available

  • Brain and Spine institute Paris

    Paris, 75013
    France

    Site Not Available

  • Charite University Medicine Berlin

    Berlin, 10117
    Germany

    Site Not Available

  • Ruhr-Univ. Bochum St. Joseph-Hospital

    Bochum, 44791
    Germany

    Site Not Available

  • George-Huntington-Institut

    Münster, 48149
    Germany

    Site Not Available

  • Ulm University, UKU, Dep. of Neurology

    Ulm, 89081
    Germany

    Site Not Available

  • Irccs Istituto Delle Scienze Neurologiche Di Bologna Uoc Clinica Neurologica

    Bologna, 40139
    Italy

    Site Not Available

  • IRCCS Carlo Besta Neurological Institutte

    Milan, 20133
    Italy

    Site Not Available

  • IRCCS Casa Sollievo della Sofferenza Research Hospital

    San Giovanni Rotondo, 71013
    Italy

    Site Not Available

  • Leiden University Medical Center

    Leiden, 2333 ZA
    Netherlands

    Site Not Available

  • University of Otago, New Zealand Brain Research Institute

    Christchurch, 8011
    New Zealand

    Site Not Available

  • Hospital Universitario Cruces

    Barakaldo, 48903
    Spain

    Site Not Available

  • Hospital de la Santa Creu I Sant Pau

    Barcelona, 8025
    Spain

    Site Not Available

  • Hospital Universitario Burgos

    Burgos, 90550
    Spain

    Site Not Available

  • Hospital Ramón y Cajal

    Madrid, 28034
    Spain

    Site Not Available

  • The Barbery National Centre for Mental Health

    Birmingham, B15 2FG
    United Kingdom

    Site Not Available

  • University Hospital of Wales

    Cardiff, CF14 4XW
    United Kingdom

    Site Not Available

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

    London, WC1N 3BG
    United Kingdom

    Site Not Available

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

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