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New drug votoplam enters final phase 3 trial for Huntington's disease

NCT ID NCT07326709

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Sep 01, 2026 · Updated 9 times

Summary

This Phase 3 trial tests whether the drug votoplam can slow the progression of Huntington's disease in people with early symptoms. About 770 adults aged 21 to 70 with a confirmed genetic diagnosis will receive either votoplam or a placebo. The main goal is to see if votoplam preserves motor, thinking, and daily function over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
votoplam (also known as HTT227, formerly PTC518)
What this could lead to
If it works, votoplam could slow the worsening of Huntington's disease, helping people maintain their motor skills, thinking, and daily function for longer.
What could go wrong
This is still an experimental drug. Even though it's in Phase 3, it may not slow the disease as hoped, and side effects are possible. Results from this large trial are needed to know if it's safe and effective.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 770 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2026

Expected to finish

Apr 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

21 to 70 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Huntington disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Huntington disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    82 sites in 22 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Albany Medical College

    RECRUITING

    Albany, New York, 12208, United States

  • Beth Israel Deaconess Med Ctr

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • CUSM Montreal Neurological Institute

    RECRUITING

    Montreal, Quebec, H3A 2B4, Canada

  • CenExcel Rocky Mtn Clin Research

    RECRUITING

    Englewood, Colorado, 80113, United States

  • Centre de recherche du CHUM

    RECRUITING

    Montreal, Quebec, H2W 1T8, Canada

  • Cleveland Clinic Foundation

    RECRUITING

    Cleveland, Ohio, 44195, United States

  • Columbia University Irving Medical

    RECRUITING

    New York, New York, 10032, United States

  • Duke University Health System

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Georgetown University

    RECRUITING

    Washington D.C., District of Columbia, 20007, United States

  • Grady Memorial Hospital and Clinics

    RECRUITING

    Atlanta, Georgia, 30303, United States

  • Henry Ford Health System

    RECRUITING

    Detroit, Michigan, 48202, United States

  • Indiana University Health

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Inje Univesrity Busan Paik Hospital

    RECRUITING

    Busan, 47392, South Korea

  • North York General Hospital

    RECRUITING

    North York, Ontario, M2K1E1, Canada

  • Novartis Investigative Site

    RECRUITING

    CABA, Buenos Aires, 1425, Argentina

  • Novartis Investigative Site

    RECRUITING

    CABA, Buenos Aires, C1056ABI, Argentina

  • Novartis Investigative Site

    RECRUITING

    Ciudad de, Buenos Aires, C1425DND, Argentina

  • Novartis Investigative Site

    RECRUITING

    Westmead, New South Wales, 2145, Australia

  • Novartis Investigative Site

    RECRUITING

    Caulfield South, Victoria, 3162, Australia

  • Novartis Investigative Site

    RECRUITING

    Leuven, Vlaams Brabant, 3000, Belgium

  • Novartis Investigative Site

    RECRUITING

    Sofia, 1431, Bulgaria

  • Novartis Investigative Site

    RECRUITING

    Guangzhou, Guangdong, 510080, China

  • Novartis Investigative Site

    RECRUITING

    Guangzhou, Guangdong, 510120, China

  • Novartis Investigative Site

    RECRUITING

    Prague, 128 08, Czechia

  • Novartis Investigative Site

    RECRUITING

    Marseille, 13885, France

  • Novartis Investigative Site

    RECRUITING

    Montpellier, 34090, France

  • Novartis Investigative Site

    RECRUITING

    Bochum, 44791, Germany

  • Novartis Investigative Site

    RECRUITING

    Budapest, 1083, Hungary

  • Novartis Investigative Site

    RECRUITING

    Bunkyo Ku, Tokyo, 1138655, Japan

  • Novartis Investigative Site

    RECRUITING

    Leiden, South Holland, 2333 ZA, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Bucharest, 040215, Romania

  • Novartis Investigative Site

    RECRUITING

    Târgu Mureş, 540136, Romania

  • Novartis Investigative Site

    RECRUITING

    Riyadh, 11211, Saudi Arabia

  • Novartis Investigative Site

    RECRUITING

    Košice, 041 90, Slovakia

  • Novartis Investigative Site

    RECRUITING

    Daejeon, Korea, 35015, South Korea

  • Novartis Investigative Site

    RECRUITING

    Barcelona, 08035, Spain

  • Novartis Investigative Site

    RECRUITING

    Barcelona, 08041, Spain

  • Novartis Investigative Site

    RECRUITING

    Seville, 41009, Spain

  • Novartis Investigative Site

    RECRUITING

    Basel, 4031, Switzerland

  • Novartis Investigative Site

    RECRUITING

    Muri bei Bern, 3073, Switzerland

  • Novartis Investigative Site

    RECRUITING

    Changhua, 50006, Taiwan

  • Novartis Investigative Site

    RECRUITING

    Taipei, 11217, Taiwan

  • Novartis Investigative Site

    RECRUITING

    Exeter, Devon, EX2 5DW, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    Aberdeen, Grampian Region, AB25 2ZN, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    Glasgow, EH9 1LF, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    London, SW17 0QT, United Kingdom

  • Novartis Investigative Site

    RECRUITING

    Oxford, OX3 9DU, United Kingdom

  • Parkinsons-Movement Disorder Ctr

    RECRUITING

    Chicago, Illinois, 60611, United States

  • SMG SNU Boramae Medical Center

    RECRUITING

    Dongjak Gu, Seoul, 07061, South Korea

  • Seoul National University Hospital

    RECRUITING

    Seoul, Seoul, 03080, South Korea

  • Southampton General Hospital

    RECRUITING

    Southampton, SO16 6YD, United Kingdom

  • Stanford Healthcare

    RECRUITING

    Stanford, California, 94305-5152, United States

  • TX Movement Disorder Spec PLLC

    RECRUITING

    Georgetown, Texas, 78628, United States

  • Tel Aviv Sourasky Med Ctr Ichilov

    RECRUITING

    Tel Aviv, 6423906, Israel

  • The Second Affiliated Hosp Of Zhejiang Univ School Of Medicine

    RECRUITING

    Hangzhou, Zhejiang, 310002, China

  • The Second Xiangya Hospital of Central South University

    RECRUITING

    Changsha, Hunan, 410011, China

  • The University of Vermont MC

    RECRUITING

    Burlington, Vermont, 05401, United States

  • Tongji Hos Affiliated Tongji Medical College of Huazhong

    RECRUITING

    Wuhan, Hubei, 430030, China

  • Trinity Health Michigan

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • U Conn Health

    RECRUITING

    Farmington, Connecticut, 06030, United States

  • U of TX Health Science Ct

    RECRUITING

    Houston, Texas, 77030, United States

  • UBMD Neurology

    RECRUITING

    Buffalo, New York, 14202, United States

  • UC Davis Health System

    RECRUITING

    Sacramento, California, 95817, United States

  • Uni Of Wisconsin Hosp And Clinic

    RECRUITING

    Madison, Wisconsin, 53705-3611, United States

  • Univ of Arkansas for Med Sciences

    RECRUITING

    Little Rock, Arkansas, 72205, United States

  • Universi of Rochester School of Med

    RECRUITING

    Rochester, New York, 14609, United States

  • University College Hospital

    RECRUITING

    London, NW1 2BU, United Kingdom

  • University Of NC At Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27599-9500, United States

  • University Of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15213, United States

  • University Of South Florida

    RECRUITING

    Tampa, Florida, 33612, United States

  • University of Alabama Birmingham

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • University of CA San Diego

    RECRUITING

    La Jolla, California, 92037, United States

  • University of Iowa Health Care

    RECRUITING

    Iowa City, Iowa, 52242-1091, United States

  • University of Kansas Hospital

    RECRUITING

    Fairway, Kansas, 66205, United States

  • University of Pennsylvania

    RECRUITING

    Philadelphia, Pennsylvania, 19107, United States

  • University of Washington Medical Center

    RECRUITING

    Seattle, Washington, 98195, United States

  • VCU Health Systems MCV

    RECRUITING

    Richmond, Virginia, 23298, United States

  • Vanderbilt University Medical CenterX

    RECRUITING

    Nashville, Tennessee, 37221, United States

  • Veracity Neuroscience LLC

    RECRUITING

    Memphis, Tennessee, 38157, United States

  • West China Hospital of Sichuan Uni

    RECRUITING

    Chengdu, Sichuan, 610041, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.