RecruitingPhase 3NCT07326709

A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease


Sponsor

Novartis Pharmaceuticals

Enrollment

770 participants

Start Date

Mar 24, 2026

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 21 YearsMax Age: 70 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Placebo and a drug called Votoplam (blinded) for people with huntington disease. The study is currently recruiting participants at 20 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGVotoplam (blinded)

Votoplam (blinded) active treatment

DRUGPlacebo

Placebo


Locations(82)

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

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

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

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

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

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

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Novartis Investigative Site

CABA, Buenos Aires, Argentina

Novartis Investigative Site

Ciudad de, 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

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

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

Prague, Czechia

Novartis Investigative Site

Marseille, France

Novartis Investigative Site

Montpellier, France

Novartis Investigative Site

Bochum, Germany

Novartis Investigative Site

Budapest, Hungary

Tel Aviv Sourasky Med Ctr Ichilov

Tel Aviv, Israel

Novartis Investigative Site

Bunkyo Ku, Tokyo, Japan

Novartis Investigative Site

Leiden, South Holland, Netherlands

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 Univesrity Busan Paik Hospital

Busan, South Korea

Novartis Investigative Site

Barcelona, Spain

Novartis Investigative Site

Barcelona, 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

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

University College Hospital

London, United Kingdom

Novartis Investigative Site

London, United Kingdom

Novartis Investigative Site

Oxford, United Kingdom

Southampton General Hospital

Southampton, United Kingdom

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NCT07326709


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