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RECRUITING

NCT07326709

PHASE3

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

Sponsor: Novartis Pharmaceuticals

View on ClinicalTrials.gov

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

2026-07-13

Healthy Volunteers

Conditions

Huntington Disease

Interventions

DRUG

Votoplam (blinded)

Votoplam (blinded) active treatment

DRUG

Placebo

Inclusion Criteria: * Signed informed consents must be obtained prior to participation in the study * Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed Consent signature * Genetically confirmed HD diagnosis with a cytosine-adenine-guanine (CAG) repeat length of 40 or above. Participants must have prior genetic confirmation and known CAG repeat length obtained prior to screening. * Meets all of the following criteria: * UHDRS IS score ≥90 * UHDRS TFC score = 13 * UHDRS TMS score = 7-25, inclusive * CAP100 ≥ 70 Calculation: CAP = Age at study entry × (CAG length - 30) / 6.49 Exclusion Criteria: * History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD * Serologic evidence for active viral hepatitis as indicated by: * positive anti-HBc IgM * positive anti-HBc IgG confirmed by positive HBsAg and/or HBV DNA * positive HCV ab test confirmed by positive HCV RNA * Immunodeficiency diseases, including a positive human immunodeficiency virus (HIV) test result * History or current diagnosis of ECG or cardiac abnormalities indicating significant risk of safety for participants such as: * Concomitant clinically significant cardiac arrhythmias, e.g., sustained ventricular tachycardia, and clinically significant second- or third-degree AV block without a pacemaker * History of familial long QT syndrome or known family history of Torsade de Pointes * Women of childbearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral salpingectomy at least six weeks before taking study treatment. In the case of oophorectomy alone, the reproductive status of the woman needs to have been confirmed by follow-up hormone level assessment. o WOCBP are excluded unless they are using highly effective methods of contraception (failure rate \< 1% per year) while taking study treatment and for 8 months after stopping study treatment. * Pregnant or nursing (breastfeeding) women Other protocol defined inclusion/exclusion criteria may apply

Locations (41)

University of CA San Diego

La Jolla, California, United States

CenExcel Rocky Mtn Clin Research

Englewood, Colorado, United States

Georgetown University

Washington D.C., District of Columbia, United States

University Of South Florida

Tampa, Florida, 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

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

Cleveland Clinic Foundation

Cleveland, Ohio, United States

University of Pennsylvania

Philadelphia, 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

University of Washington Medical Center

Seattle, Washington, United States

Novartis Investigative Site

CABA, Buenos Aires, Argentina

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

Chengdu, Sichuan, China

Tel Aviv Sourasky Med Ctr Ichilov

Tel Aviv, Israel

Novartis Investigative Site

Dongjak Gu, Seoul, South Korea

Novartis Investigative Site

Seoul, Seoul, South Korea

Novartis Investigative Site

Busan, South Korea

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

Cardiff, United Kingdom

Novartis Investigative Site

Glasgow, United Kingdom

University College Hospital

London, United Kingdom

Novartis Investigative Site

Oxford, United Kingdom

Southampton General Hospital

Southampton, United Kingdom

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (41)