Actively Recruiting
A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Led by Novartis Pharmaceuticals · Updated on 2026-05-14
770
Participants Needed
10
Research Sites
214 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official 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 prior to participation
- Ambulatory males or females aged 21 to 70 years inclusive at consent
- Genetically confirmed Huntington's disease with CAG repeat length of 40 or more
- UHDRS Independence Scale score of 90 or less
- UHDRS Total Functional Capacity score equal to 13
- UHDRS Total Motor Score between 7 and 25 inclusive
- CAP score 100 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
- Positive tests indicating active viral hepatitis infection
- Immunodeficiency diseases including positive HIV test
- Significant cardiac abnormalities such as clinically significant arrhythmias or second- or third-degree AV block without pacemaker
- History of familial long QT syndrome or family history of Torsade de Pointes
- Women of childbearing potential unless surgically sterile or using highly effective contraception during and for 8 months after treatment
- Pregnant or breastfeeding women
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 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
U of TX Health Science Ct
Houston, Texas, United States, 77030
Actively Recruiting
6
University of Washington Medical Center
Seattle, Washington, United States, 98195
Actively Recruiting
7
North York General Hospital
North York, Ontario, Canada, M2K1E1
Actively Recruiting
8
Centre de recherche du CHUM
Montreal, Quebec, Canada, H2W 1T8
Actively Recruiting
9
Novartis Investigative Site
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
10
Novartis Investigative Site
London, United Kingdom, NW1 2BU
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
CONTACT
N
Novartis Pharmaceuticals
CONTACT
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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