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A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Sponsor: Novartis Pharmaceuticals
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.
Official title: A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Key Details
Gender
All
Age Range
21 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
770
Start Date
2026-03-24
Completion Date
2030-04-30
Last Updated
2026-09-22
Healthy Volunteers
No
Conditions
Interventions
Votoplam (blinded)
Votoplam (blinded) active treatment
Placebo
Placebo
Locations (117)
University of Alabama Birmingham
Birmingham, Alabama, United States
Univ of Arkansas for Med Sciences
Little Rock, Arkansas, United States
University of CA San Diego
La Jolla, California, United States
UC Davis Health System
Sacramento, California, United States
UCSF
San Francisco, California, United States
Stanford Healthcare
Stanford, California, United States
CenExcel Rocky Mtn Clin Research
Englewood, Colorado, United States
U Conn Health
Farmington, Connecticut, United States
Georgetown University
Washington D.C., District of Columbia, United States
UM Department Of Neurology
Miami, Florida, United States
University Of South Florida
Tampa, Florida, United States
Grady Memorial Hospital and Clinics
Atlanta, Georgia, United States
Parkinsons-Movement Disorder Ctr
Chicago, Illinois, United States
Rush University Medical Center
Chicago, Illinois, United States
Indiana University Health
Indianapolis, Indiana, United States
University of Iowa Health Care
Iowa City, Iowa, United States
University of Kansas Hospital
Fairway, Kansas, United States
Hereditary Neurological Disease Centre
Wichita, Kansas, United States
Johns Hopkins University
Baltimore, Maryland, United States
Beth Israel Deaconess Med Ctr
Boston, Massachusetts, United States
Henry Ford Health System
Detroit, Michigan, United States
Trinity Health Michigan
Grand Rapids, Michigan, United States
Albany Medical College
Albany, New York, United States
UBMD Neurology
Buffalo, New York, United States
Columbia University Irving Medical
New York, New York, United States
Universi of Rochester School of Med
Rochester, New York, United States
University Of NC At Chapel Hill
Chapel Hill, North Carolina, United States
Duke University Health System
Durham, North Carolina, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Oregon Health Sciences University
Portland, Oregon, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University Of Pittsburgh
Pittsburgh, Pennsylvania, United States
Veracity Neuroscience LLC
Memphis, Tennessee, United States
Vanderbilt University Medical CenterX
Nashville, Tennessee, United States
TX Movement Disorder Spec PLLC
Georgetown, Texas, United States
U of TX Health Science Ct
Houston, Texas, United States
The University of Vermont MC
Burlington, Vermont, United States
VCU Health Systems MCV
Richmond, Virginia, United States
University of Washington Medical Center
Seattle, Washington, United States
Uni Of Wisconsin Hosp And Clinic
Madison, Wisconsin, United States
Instituto Argentino de Investigación Neurológica (IADIN)
CABA, Buenos Aires, Argentina
Centro de Investigaciones Metabólicas (CINME)
CABA, Buenos Aires, Argentina
Novartis Investigative Site
Ciudad de, Buenos Aires, Argentina
Novartis Investigative Site
Buenos Aires, Argentina
Novartis Investigative Site
Westmead, New South Wales, Australia
Novartis Investigative Site
Caulfield South, Victoria, Australia
Novartis Investigative Site
Leuven, Vlaams Brabant, Belgium
Novartis Investigative Site
Sofia, Bulgaria
Novartis Investigative Site
Sofia, Bulgaria
North York General Hospital
North York, Ontario, Canada
Centre de recherche du CHUM
Montreal, Quebec, Canada
CUSM Montreal Neurological Institute
Montreal, Quebec, Canada
Novartis Investigative Site
Fuzhou, Fujian, China
Novartis Investigative Site
Guangzhou, Guangdong, China
Novartis Investigative Site
Guangzhou, Guangdong, China
Tongji Hos Affiliated Tongji Medical College of Huazhong
Wuhan, Hubei, China
The Second Xiangya Hospital of Central South University
Changsha, Hunan, China
West China Hospital of Sichuan Uni
Chengdu, Sichuan, China
The Second Affiliated Hosp Of Zhejiang Univ School Of Medicine
Hangzhou, Zhejiang, China
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Shanghai, China
Novartis Investigative Site
Medellín, Colombia
Novartis Investigative Site
Brno, Czechia
Novartis Investigative Site
Prague, Czechia
Novartis Investigative Site
Copenhagen, Denmark
Novartis Investigative Site
Angers, France, France
Novartis Investigative Site
Marseille, France
Novartis Investigative Site
Montpellier, France
Novartis Investigative Site
Aachen, Germany
Novartis Investigative Site
Bochum, Germany
Novartis Investigative Site
Münster, Germany
Novartis Investigative Site
Tübingen, Germany
Novartis Investigative Site
Ulm, Germany
Novartis Investigative Site
Budapest, Hungary
Novartis Investigative Site
Szeged, Hungary
Tel Aviv Sourasky Med Ctr Ichilov
Tel Aviv, Israel
Novartis Investigative Site
Bologna, BO, Italy
Novartis Investigative Site
Brescia, BS, Italy
Novartis Investigative Site
Milan, MI, Italy
Novartis Investigative Site
Milan, MI, Italy
Novartis Investigative Site
Sagamihara, Kanagawa, Japan
Novartis Investigative Site
Kashihara, Nara, Japan
Novartis Investigative Site
Bunkyo Ku, Tokyo, Japan
Novartis Investigative Site
Maastricht, Limburg, Netherlands
Novartis Investigative Site
Leiden, South Holland, Netherlands
Novartis Investigative Site
Krakow, POL, Poland
Novartis Investigative Site
Lisbon, Portugal
Novartis Investigative Site
Porto, Portugal
Novartis Investigative Site
Bucharest, Romania
Novartis Investigative Site
Târgu Mureş, Romania
Novartis Investigative Site
Riyadh, Saudi Arabia
Novartis Investigative Site
Košice, Slovakia
Novartis Investigative Site
Daejeon, Korea, South Korea
SMG SNU Boramae Medical Center
Dongjak Gu, Seoul, South Korea
Seoul National University Hospital
Seoul, Seoul, South Korea
Inje University Busan Paik Hospital
Busan, South Korea
Novartis Investigative Site
Palma, Balearic Islands, Spain
Novartis Investigative Site
Barcelona, Spain
Novartis Investigative Site
Barcelona, Spain
Novartis Investigative Site
Madrid, Spain
Novartis Investigative Site
Seville, Spain
Novartis Investigative Site
Basel, Switzerland
Novartis Investigative Site
Muri bei Bern, Switzerland
Novartis Investigative Site
Changhua, Taiwan
Novartis Investigative Site
Taipei, Taiwan
Novartis Investigative Site
Taoyuan, Taiwan
Novartis Investigative Site
Exeter, Devon, United Kingdom
Novartis Investigative Site
Aberdeen, Grampian Region, United Kingdom
Novartis Investigative Site
Cambridge, United Kingdom
Novartis Investigative Site
Cardiff, United Kingdom
Novartis Investigative Site
Glasgow, United Kingdom
Novartis Investigative Site
Leeds, United Kingdom
University College Hospital
London, United Kingdom
Novartis Investigative Site
London, United Kingdom
Novartis Investigative Site
Oxford, United Kingdom
Southampton General Hospital
Southampton, United Kingdom