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

NCT ID NCT07326709

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

First seen Jun 25, 2026 · Last updated Aug 12, 2026 · Updated 7 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.

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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

Contacts and locations

Locations

Albany Medical College
RECRUITING

Albany, New York, 12208, 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
Henry Ford Health System
RECRUITING

Detroit, Michigan, 48202, United States
Indiana University Health
RECRUITING

Indianapolis, Indiana, 46202, United States
Inje University 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

Caulfield South, Victoria, 3162, Australia
Novartis Investigative Site
RECRUITING

Leuven, Vlaams Brabant, 3000, Belgium
Novartis Investigative Site
RECRUITING

Wuhan, Hubei, 430030, China
Novartis Investigative Site
RECRUITING

Changsha, Hunan, 410011, China
Novartis Investigative Site
RECRUITING

Hangzhou, Zhejiang, 310002, China
Novartis Investigative Site
RECRUITING

Montpellier, 34090, France
Novartis Investigative Site
RECRUITING

Bunkyo Ku, Tokyo, 113-8655, Japan
Novartis Investigative Site
RECRUITING

Daejeon, Korea, 35015, South Korea
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

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 University of Vermont MC
RECRUITING

Burlington, Vermont, 05401, United States
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
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 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
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

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