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RECRUITING Phase 3 ClinicalTrials.gov

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

Sponsor: Novartis Pharmaceuticals • ClinicalTrials.gov ID: NCT07326709

Registry updated: 2026-09-22
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Description

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.

Interventions

  • Votoplam (blinded) (Drug)
    Votoplam (blinded) active treatment
  • Placebo (Drug)
    Placebo

Eligibility criteria

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

Conditions

Huntington Disease

Locations

  • 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
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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

View Official Record