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A Study of Votoplam for Huntington's Disease

This study is looking into a drug called Votoplam to see if it is safe and if it can slow down the progression of Huntington's disease (HD) in people who are showing early symptoms. Researchers will compare Votoplam to a placebo (an inactive substance that looks like the study drug). You might be able to join if you are between 21 and 70 years old, can walk, and have a confirmed genetic diagnosis of HD with a specific genetic marker (CAG repeat length of 40 or above). The main way they will measure success is by looking at changes in your cUHDRS score, which is a way to measure HD symptoms, over 36 months. The study plans to enroll about 770 participants, but its current recruitment status is unclear.

Study design
This is an interventional study where participants will receive either Votoplam or a placebo, and neither you nor your doctor will know which one you are receiving (this is called 'blinded'). The treatment period can last up to 36 months.
What's involved
You would first go through a screening period of up to 42 days to see if you qualify. If eligible, you would then be in the double-blind treatment period for up to 36 months. Not every participant will complete 36 months of treatment.
Compensation
Not stated in the trial record.
Follow-up
After your treatment period ends, there will be one safety follow-up visit, either in person or by phone, about 30 days later.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07326709

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

Recruiting
PHASE3Ages 21–70InterventionalTreatment
Novartis Pharmaceuticals
~770 participants
Updated 2026-08-31 on ClinicalTrials.gov
What's tested:Votoplam (blinded)Placebo

At a glance

Recruiting sites
82 of 82 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change from Baseline in cUHDRS score
Measured over Baseline, Month 36

Conditions

Huntington Disease

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NCT07326709

Where you'd take part

This study runs at 82 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Albany Medical College

    Albany, New Yorkstudy coordinator listed

    Recruiting

  • Beth Israel Deaconess Med Ctr

    Boston, Massachusettsstudy coordinator listed

    Recruiting

  • CenExcel Rocky Mtn Clin Research

    Englewood, Coloradostudy coordinator listed

    Recruiting

  • Centre de recherche du CHUM

    Montreal, Quebec, Canadastudy coordinator listed

    Recruiting

  • Cleveland Clinic Foundation

    Cleveland, Ohiostudy coordinator listed

    Recruiting

  • Columbia University Irving Medical

    New York, New Yorkstudy coordinator listed

    Recruiting

  • CUSM Montreal Neurological Institute

    Montreal, Quebec, Canadastudy coordinator listed

    Recruiting

  • Duke University Health System

    Durham, North Carolinastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Who to contact

Novartis Pharmaceuticals
[email protected]
1-888-669-6682
Email the study team

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Eligibility criteria

Inclusion

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

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.
Pregnant or nursing (breastfeeding) women

What this trial measures

  • Change from Baseline in cUHDRS scoreBaseline, Month 36

    The Composite Unified Huntingtons Disease Ratings Scale (cUHDRS) is an improved composite measure to assess multi-domain clinical progression in HD. The cUHDRS is a combined weighted score of measures of motor function (TMS), cognition (SDMT and SWRT) and overall functional capacity (TFC): cUHDRS = \[(TFC-10.4)/1.9 - (TMS-29.7)/14.9 + (SDMT-28.4)/11.3 + (SWRT - 66.1)/20.1\] + 10. In conjunction with one another, these measures provide a comprehensive, sensitive and specific tool for monitoring disease progression, with lower scores indicating more severe disease. cUHDRS score range from -8 to 25.