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

Last updated: November 18, 2025
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Not Recruiting

Phase

2

Condition

Dyskinesias

Holoprosencephaly

Neuronal Ceroid Lipofuscinoses (Ncl)

Treatment

PTC518

Votoplam

Clinical Study ID

NCT06254482
PTC518-CNS-004-HD
CHTT227A12201
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 votoplam 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: 144
Treatment Group(s): 2
Primary Treatment: PTC518
Phase: 2
Study Start date:
August 25, 2023
Estimated Completion Date:
July 16, 2029

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 votoplam in this extension study. Participants who received votoplam 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 milligrams [mg]). Participants who received placebo in the parent Study PTC518-CNS-002-HD will be allocated to a votoplam dose level according to the same dosing group in which they were previously randomized (5, 10 mg).

Connect with a study center

  • Monash Health

    Clayton, 3168
    Australia

    Site Not Available

  • Novartis Investigative Site

    Clayton 2171400, 3168
    Australia

    Site Not Available

  • Westmead Hospital

    Westmead, 2145
    Australia

    Site Not Available

  • Novartis Investigative Site

    Westmead 2143973, 2145
    Australia

    Site Not Available

  • Medical University Innsbruck

    Innsbruck, 6020
    Austria

    Site Not Available

  • Novartis Investigative Site

    Innsbruck 2775220, 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

  • Novartis Investigative Site

    Ottawa 6094817, 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

  • Novartis Investigative Site

    Angers 3037656, 49100
    France

    Site Not Available

  • Hôpital Universitaire de Marseille Hôpital de la Timone

    Marseille, 13385
    France

    Site Not Available

  • Novartis Investigative Site

    Marseille 2995469, 13385
    France

    Site Not Available

  • Brain and Spine institute Paris

    Paris, 75013
    France

    Site Not Available

  • Novartis Investigative Site

    Paris 2988507, 75013
    France

    Site Not Available

  • Charite University Medicine Berlin

    Berlin, 10117
    Germany

    Site Not Available

  • Novartis Investigative Site

    Berlin 2950159, 10117
    Germany

    Site Not Available

  • Ruhr-Univ. Bochum St. Joseph-Hospital

    Bochum, 44791
    Germany

    Site Not Available

  • Novartis Investigative Site

    Bochum 2947416, 44791
    Germany

    Site Not Available

  • George-Huntington-Institut

    Münster, 48149
    Germany

    Site Not Available

  • Novartis Investigative Site

    Münster 2867543, 48149
    Germany

    Site Not Available

  • Ulm University, UKU, Dep. of Neurology

    Ulm, 89081
    Germany

    Site Not Available

  • Novartis Investigative Site

    Ulm 2820256, 89081
    Germany

    Site Not Available

  • Irccs Istituto Delle Scienze Neurologiche Di Bologna Uoc Clinica Neurologica

    Bologna, 40139
    Italy

    Site Not Available

  • Novartis Investigative Site

    Bologna 3181928, 40139
    Italy

    Site Not Available

  • IRCCS Carlo Besta Neurological Institutte

    Milan, 20133
    Italy

    Site Not Available

  • Novartis Investigative Site

    Milan 3173435, 20133
    Italy

    Site Not Available

  • IRCCS Casa Sollievo della Sofferenza Research Hospital

    San Giovanni Rotondo, 71013
    Italy

    Site Not Available

  • Novartis Investigative Site

    San Giovanni Rotondo 3168234, 71013
    Italy

    Site Not Available

  • Leiden University Medical Center

    Leiden, 2333 ZA
    Netherlands

    Site Not Available

  • Novartis Investigative Site

    Leiden 2751773, 2333 ZA
    Netherlands

    Site Not Available

  • University of Otago, New Zealand Brain Research Institute

    Christchurch, 8011
    New Zealand

    Site Not Available

  • Novartis Investigative Site

    Christchurch 2192362, 8011
    New Zealand

    Site Not Available

  • Hospital Universitario Cruces

    Barakaldo, 48903
    Spain

    Site Not Available

  • Novartis Investigative Site

    Barakaldo 3109453, 48903
    Spain

    Site Not Available

  • Hospital de la Santa Creu I Sant Pau

    Barcelona, 8025
    Spain

    Site Not Available

  • Novartis Investigative Site

    Barcelona 3128760, 8025
    Spain

    Site Not Available

  • Hospital Universitario Burgos

    Burgos, 90550
    Spain

    Site Not Available

  • Novartis Investigative Site

    Burgos 3127461, 90550
    Spain

    Site Not Available

  • Hospital Ramón y Cajal

    Madrid, 28034
    Spain

    Site Not Available

  • Novartis Investigative Site

    Madrid 3117735, 28034
    Spain

    Site Not Available

  • The Barbery National Centre for Mental Health

    Birmingham, B15 2FG
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    Birmingham 2655603, B15 2FG
    United Kingdom

    Site Not Available

  • University Hospital of Wales

    Cardiff, CF14 4XW
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    Cardiff 2653822, 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

  • Novartis Investigative Site

    London 2643743, WC1N 3BG
    United Kingdom

    Site Not Available

  • Manchester University NHS Foundation Trust

    Manchester, M13 9WL
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    Manchester 2643123, M13 9WL
    United Kingdom

    Site Not Available

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