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A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Sponsor: Novartis Pharmaceuticals
Summary
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.
Official title: A Phase 2A, Randomized, Placebo-Controlled, Dose-Ranging Study to Evaluate the Safety and Efficacy of PTC518 in Subjects With Huntington's Disease
Key Details
Gender
All
Age Range
25 Years - Any
Study Type
INTERVENTIONAL
Enrollment
159
Start Date
2022-06-03
Completion Date
2025-07-31
Last Updated
2026-06-16
Healthy Volunteers
No
Conditions
Interventions
PTC518
PTC518 will be administered per dose and schedule specified in the arm.
Placebo
Placebo matching to PTC518 will be administered per schedule specified in the arm.
Locations (28)
University of California, San Diego
La Jolla, California, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
The University of Texas Health Science Center at Houston; McGovern Medical School
Houston, Texas, United States
University of Washington Department of Neurology
Seattle, Washington, United States
Monash Health
Clayton, Australia
Westmead Hospital
Sydney, Australia
Medical University Innsbruck
Innsbruck, Austria
The Ottawa Hospital, Parkinson's and Movement Disorders Clinic
Ottawa, Canada
Centre Hospitalier Universitaire d'Angers
Angers, France
Hôpital Universitaire de Marseille Hôpital de la Timone
Marseille, France
Brain and Spine Institute Paris
Paris, France
Charite University Medicine Berlin
Berlin, Germany
Ruhr-Univ. Bochum St. Joseph-Hospital
Bochum, Germany
George-Huntington-Institut
Münster, Germany
Ulm University, UKU, Dep. of Neurology
Ulm, Germany
Irccs Istituto Delle Scienze Neurologiche Di Bologna Uoc Clinica Neurologica
Bologna, Italy
IRCCS Carlo Besta Neurological Institutte
Milan, Italy
IRCCS Casa Sollievo della Sofferenza Research Hospital
San Giovanni Rotondo, Italy
Leiden University Medical Center
Leiden, Netherlands
University of Otago, New Zealand Brain Research Institute
Christchurch, New Zealand
Hospital Universitario Cruces
Barakaldo, Spain
Hospital de la Santa Creu I Sant Pau
Barcelona, Spain
Hospital Universitario Burgos
Burgos, Spain
Hospital Ramón y Cajal
Madrid, Spain
The Barbery National Centre for Mental Health
Birmingham, United Kingdom
Cardiff University Schools of Medicine and Biosciences
Cardiff, United Kingdom
UCL Queen Square Institute of Neurology National Hospital for Neurology and Neurosurgery
London, United Kingdom
Manchester University NHS Foundation Trust
Manchester, United Kingdom