A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Start Date
3/24/2026
Completion Date
4/30/2030
Summary
The purpose is to assess safety and tolerability of votoplam and to determine whether votoplam slows disease progression in patients with early symptomatic Huntington's disease (HD) compared to the control arm. HTT227 - current compound code (former code is PTC518 from PTC Therapeutics), HTT227 is Novartis code under Novartis sponsorship.
Detailed Description
This study will have a variable double-blind treatment duration of up to 36 months. As part of the study design, not every participant will complete 36 months of treatment. The study consists of 3 periods: * Screening Period: A period of up to 42-days to assess participants eligibility * Double-blind Treatment Period: This period will have variable individual treatment duration, up to 36 months. The double-blind treatment period concludes when ≥50% patients complete Month 36. The maximum treatment duration for an individual participant is 36 months. * Safety Follow-up Period: A period consisting of one safety follow-up visit, conducted on site or by phone call, for all participants not continuing treatment in the separate open-label extension study or discontinuing early. The visit/phone call will take place 30 days after End of Study (EOS)
Eligibility Criteria
Age Range: 21 years to 70 years
Interventions
Votoplam (blinded)
Placebo
Conditions
Locations
University of CA San Diego
La Jolla, California 92037
United States
Stanford Healthcare
Stanford, California 94305 5152
United States
CenExcel Rocky Mtn Clin Research
Englewood, Colorado 80113
United States
Georgetown University
Washington D.C., District of Columbia 20007
United States
University Of South Florida
Tampa, Florida 33612
United States
Parkinsons-Movement Disorder Ctr
Chicago, Illinois 60611
United States
Indiana University Health
Indianapolis, Indiana 46202
United States
University of Iowa Health Care
Iowa City, Iowa 52242-1091
United States
University of Kansas Hospital
Fairway, Kansas 66205
United States
Henry Ford Health System
Detroit, Michigan 48202
United States
Trinity Health Michigan
Grand Rapids, Michigan 49503
United States
Albany Medical College
Albany, New York 12208
United States
UBMD Neurology
Buffalo, New York 14202
United States
Columbia University Irving Medical
New York, New York 10032
United States
Universi of Rochester School of Med
Rochester, New York 14609
United States
University Of NC At Chapel Hill
Chapel Hill, North Carolina 27599 9500
United States
Cleveland Clinic Foundation
Cleveland, Ohio 44195
United States
University of Pennsylvania
Philadelphia, Pennsylvania 19107
United States
Veracity Neuroscience LLC
Memphis, Tennessee 38157
United States
Vanderbilt University Medical CenterX
Nashville, Tennessee 37221
United States
TX Movement Disorder Spec PLLC
Georgetown, Texas 78628
United States
U of TX Health Science Ct
Houston, Texas 77030
United States
The University of Vermont MC
Burlington, Vermont 05401
United States
University of Washington Medical Center
Seattle, Washington 98195
United States
Uni Of Wisconsin Hosp And Clinic
Madison, Wisconsin 53705-3611
United States
Novartis Investigative Site
CABA, Buenos Aires 1425
Argentina
Novartis Investigative Site
CABA, Buenos Aires C1056ABI
Argentina
Novartis Investigative Site
Caulfield South, Victoria 3162
Australia
North York General Hospital
North York, Ontario M2K1E1
Canada
Centre de recherche du CHUM
Montreal, Quebec H2W 1T8
Canada
CUSM Montreal Neurological Institute
Montreal, Quebec H3A 2B4
Canada
West China Hospital of Sichuan Uni
Chengdu, Sichuan 610041
China
Novartis Investigative Site
Hangzhou, Zhejiang 310002
China
Novartis Investigative Site
Bordeaux, 33076
France
Novartis Investigative Site
Montpellier, 34090
France
Tel Aviv Sourasky Med Ctr Ichilov
Tel Aviv, 6423906
Israel
Novartis Investigative Site
Dongjak Gu, Seoul 07061
South Korea
Novartis Investigative Site
Seoul, Seoul 03080
South Korea
Novartis Investigative Site
Busan, 47392
South Korea
Novartis Investigative Site
Basel, 4031
Switzerland
Novartis Investigative Site
Muri bei Bern, 3073
Switzerland
Novartis Investigative Site
Changhua, 50006
Taiwan
Novartis Investigative Site
Taipei, 11217
Taiwan
Novartis Investigative Site
Exeter, Devon EX2 5DW
United Kingdom
Novartis Investigative Site
Aberdeen, Grampian Region AB25 2ZN
United Kingdom
Novartis Investigative Site
Cambridge, CB2 0QQ
United Kingdom
Novartis Investigative Site
Cardiff, CF14 4XW
United Kingdom
Novartis Investigative Site
Glasgow, EH9 1LF
United Kingdom
University College Hospital
London, NW1 2BU
United Kingdom
Novartis Investigative Site
Oxford, OX3 9DU
United Kingdom
Southampton General Hospital
Southampton, SO16 6YD
United Kingdom