Testing Votoplam (blinded) against a placebo for huntington disease
Official title: A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
- Phase 3
- 2 groups
- 2 sites in Montreal
- Recruiting
Interventions
- Medication
Votoplam (blinded)
Votoplam (blinded) active treatment
- Medication
Placebo
Placebo
Canadian Sites (3)
3 of 3 recruiting
- Recruiting
North York General Hospital
North York, Ontario
- Recruiting
Centre de recherche du CHUM
Montreal, Quebec
- Recruiting
CUSM Montreal Neurological Institute
Montreal, Quebec
Eligibility Criteria
See who this study is looking for21 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Ambulatory male or female participants between 21 to 70 years of age, inclusive, on the day of Informed ConsentInformed consentThe process of being told what taking part involves, then choosing freely.Read more → signature
- +UHDRS TMS score = 7-25, inclusive
- +Signed informed consents must be obtained prior to participation in the study
- +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 screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- +Meets all of the following criteria:
- +UHDRS IS score ≥90
- +UHDRS TFC score = 13
- +CAP100 ≥ 70 Calculation: CAP = Age at study entry × (CAG length - 30) / 6.49
Exclusion
- −Serologic evidence for active viral hepatitis as indicated by:
- −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
- −positive anti-HBc IgM
- −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
- −Other protocolProtocolThe detailed plan a study must follow.Read more → defined inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →/exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → may apply
- −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-upFollow-upContinued check-ins after the treatment part is finished.Read more → 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
- −History of gene therapy or cell transplantation or any other experimental brain surgery for the treatment of HD
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more → — a large study comparing this against the current standard, usually across many hospitals.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 2 groups in this study.
Group A receives Votoplam (blinded).
Registry label: A: Votoplam
Group B, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receives Placebo.
Registry label: B: Placebo
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 21 years to 70 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 770 people.
The study is currently expected to finish around April 2030.
The main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more →, Month 36.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Change from BaselineBaselineYour starting measurements, taken before treatment begins.Read more → in cUHDRS score — measured over Baseline, Month 36.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07326709 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About Votoplam (blinded)Drug
Votoplam (blinded) active treatment
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About PlaceboDrug
Placebo
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 21 years to 70 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 21 years to 70 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more →, Month 36.
The study as a whole is currently expected to finish around 2030-04-30.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 770 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 9 locations, including: North York, Ontario, Canada; Montreal, Quebec, Canada; Detroit, Michigan, United States; Grand Rapids, Michigan, United States; Albany, New York, United States; Buffalo, New York, United States, and 3 more.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
Other Sites (7)
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 Washington Medical Center
Seattle, Washington, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.