New drug votoplam enters final phase 3 trial for Huntington's disease
NCT ID NCT07326709
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Albany Medical College
RECRUITINGAlbany, New York, 12208, United States
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CUSM Montreal Neurological Institute
RECRUITINGMontreal, Quebec, H3A 2B4, Canada
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CenExcel Rocky Mtn Clin Research
RECRUITINGEnglewood, Colorado, 80113, United States
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Centre de recherche du CHUM
RECRUITINGMontreal, Quebec, H2W 1T8, Canada
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Cleveland Clinic Foundation
RECRUITINGCleveland, Ohio, 44195, United States
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Columbia University Irving Medical
RECRUITINGNew York, New York, 10032, United States
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Georgetown University
RECRUITINGWashington D.C., District of Columbia, 20007, United States
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North York General Hospital
RECRUITINGNorth York, Ontario, M2K1E1, Canada
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Novartis Investigative Site
RECRUITINGChengdu, Sichuan, 610041, China
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Novartis Investigative Site
RECRUITINGSeoul, Seoul, 03080, South Korea
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Novartis Investigative Site
RECRUITINGTaipei, 11217, Taiwan
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Novartis Investigative Site
RECRUITINGExeter, Devon, EX2 5DW, United Kingdom
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Novartis Investigative Site
RECRUITINGAberdeen, Grampian Region, AB25 2ZN, United Kingdom
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Novartis Investigative Site
RECRUITINGCardiff, CF14 4XW, United Kingdom
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Novartis Investigative Site
RECRUITINGGlasgow, G51 4TF, United Kingdom
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Parkinsons-Movement Disorder Ctr
RECRUITINGChicago, Illinois, 60611, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Southampton General Hospital
RECRUITINGSouthampton, SO16 6YD, United Kingdom
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TX Movement Disorder Spec PLLC
RECRUITINGGeorgetown, Texas, 78628, United States
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Tel Aviv Sourasky Med Ctr Ichilov
RECRUITINGTel Aviv, 6423906, Israel
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U of TX Health Science Ct
RECRUITINGHouston, Texas, 77030, United States
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UBMD Neurology
RECRUITINGBuffalo, New York, 14202, United States
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University College Hospital
RECRUITINGLondon, NW1 2BU, United Kingdom
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University of CA San Diego
RECRUITINGLa Jolla, California, 92037, United States
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University of Kansas Hospital
RECRUITINGFairway, Kansas, 66205, United States
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University of Pennsylvania
RECRUITINGPhiladelphia, Pennsylvania, 19107, United States
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University of Washington Medical Center
RECRUITINGSeattle, Washington, 98195, United States
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Veracity Neuroscience LLC
RECRUITINGMemphis, Tennessee, 38157, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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