Actively Recruiting
A Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Efficacy, Safety, and Tolerability of Votoplam in Participants With Huntington's Disease
Led by Novartis Pharmaceuticals · Updated on 2026-06-08
770
Participants Needed
20
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of votoplam in people with early symptomatic Huntington's disease (HD) to see if it slows disease progression compared to a placebo. This study is a phase 3 randomized, double-blind trial sponsored by Novartis Pharmaceuticals. The investigation focuses on whether votoplam can impact disease measures over time in adults aged 21 to 70 who have genetically confirmed HD with specific clinical scores. Participants will be randomly assigned in a 3:2 ratio to receive either votoplam or a placebo, both taken orally. The study includes three periods: a screening period of up to 42 days to determine eligibility, a double-blind treatment period lasting up to 36 months, and a safety follow-up visit about 30 days after the end of treatment or study. Not all participants will complete the full 36 months of treatment. Throughout the study, participants will undergo various assessments including clinical rating scales such as the cUHDRS and UHDRS components, blood tests for mutant huntingtin protein and neurofilament light levels, and monitoring for adverse events. The main outcome measured is the change in the cUHDRS score from baseline to month 36. Safety follow-up will be conducted in person or by phone, ensuring close monitoring of participants during and after treatment.
CONDITIONS
Brief Title
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent before joining the study
- Male or female participants aged 21 to 70 years on the day of consent
- Genetically confirmed Huntington's disease with a CAG repeat length of 40 or higher
- UHDRS Independence Scale score of 90 or more
- UHDRS Total Functional Capacity score of 13
- UHDRS Total Motor Score between 7 and 25 inclusive
- CAP score of 70 or higher calculated as Age at study entry × (CAG length - 30) / 6.49
You will not qualify if you...
- History of gene therapy, cell transplantation, or experimental brain surgery for Huntington's disease
- Active viral hepatitis indicated by specific blood tests
- Immunodeficiency diseases including positive HIV test
- Significant cardiac abnormalities such as sustained ventricular tachycardia or advanced AV block without pacemaker
- Family history of long QT syndrome or Torsade de Pointes
- Women of childbearing potential not using highly effective contraception or who are pregnant or breastfeeding
- Other protocol-defined criteria as applicable
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - Up to 6 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Variable, up to 36 months
Participants receive blinded treatment with either Votoplam or placebo taken orally.
Duration - Single visit 30 days after end of treatment
Participants who discontinue early or do not continue in the open-label extension study have one safety follow-up visit or phone call 30 days after the end of treatment.
1 visit or phone call
Trial Site Locations
Total: 20 locations
1
CenExcel Rocky Mtn Clin Research
Englewood, Colorado, United States, 80113
Actively Recruiting
2
Georgetown University
Washington D.C., District of Columbia, United States, 20007
Actively Recruiting
3
Albany Medical College
Albany, New York, United States, 12208
Actively Recruiting
4
UBMD Neurology
Buffalo, New York, United States, 14202
Actively Recruiting
5
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
Actively Recruiting
6
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19107
Actively Recruiting
7
TX Movement Disorder Spec PLLC
Georgetown, Texas, United States, 78628
Actively Recruiting
8
U of TX Health Science Ct
Houston, Texas, United States, 77030
Actively Recruiting
9
University of Washington Medical Center
Seattle, Washington, United States, 98195
Actively Recruiting
10
North York General Hospital
North York, Ontario, Canada, M2K1E1
Actively Recruiting
11
Centre de recherche du CHUM
Montreal, Quebec, Canada, H2W 1T8
Actively Recruiting
12
CUSM Montreal Neurological Institute
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
13
Novartis Investigative Site
Tel Aviv, Israel, 6423906
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14
Novartis Investigative Site
Seoul, Seoul, South Korea, 03080
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15
Novartis Investigative Site
Taipei, Taiwan, 11217
Actively Recruiting
16
Novartis Investigative Site
Aberdeen, Grampian Region, United Kingdom, AB25 2ZN
Actively Recruiting
17
Novartis Investigative Site
Cardiff, United Kingdom, CF14 4XW
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18
Novartis Investigative Site
Glasgow, United Kingdom, G51 4TF
Actively Recruiting
19
University College Hospital
London, United Kingdom, NW1 2BU
Actively Recruiting
20
Southampton General Hospital
Southampton, United Kingdom, SO16 6YD
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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