At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Votoplam (blinded) (drug), Placebo (drug)
- How long the study runs
- Study runs about 49 months (dates as stated)
- About the drug or intervention
- Votoplam (blinded) — drug: Votoplam (blinded) active treatment · Placebo — drug: Placebo
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 21 Years to 70 Years
- Who
- All
- Number of participants
- 770
- Started
- 2026-03-24
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for huntington disease
- • Phase3 - 770 participants
- • 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
Who can take part?
- • Ages 21 Years to 70 Years
- • Diagnosed with huntington disease
Where?
- • Exeter - Novartis Investigative Site
- • Aberdeen - Novartis Investigative Site
- • Cambridge - Novartis Investigative Site
- • Cardiff - Novartis Investigative Site
- • +6 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
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.
More detail
This study will have a variable double-blind treatment duration of up to 36 months. As part of the study design, not every participant will complete 36 months of treatment. The study consists of 3 periods: * Screening Period: A period of up to 42-days to assess participants eligibility * Double-blind Treatment Period: This period will have variable individual treatment duration, up to 36 months. The double-blind treatment period concludes when ≥50% patients complete Month 36. The maximum treatment duration for an individual participant is 36 months. * Safety Follow-up Period: A period consisting of one safety follow-up visit, conducted on site or by phone call, for all participants not continuing treatment in the separate open-label extension study or discontinuing early. The visit/phone call will take place 30 days after End of Study (EOS)
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 21 Years - 70 Years
- Who can join: All genders
Biomarkers mentioned
Treatment history
Treatments you must have had:
- ✓ genetic confirmation and known CAG repeat length obtained
What the study is looking for
- ✓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 agreement to take part...
- ✓Meets all of the following criteria:
- ✓UHDRS IS score ≥90
- ✓UHDRS TFC score = 13
Who cannot take part
- ✗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
See the full criteria
Where Is This Study? (10 UK sites)
Novartis Investigative Site
Exeter EX2 5DW, United Kingdom
Novartis Investigative Site
Aberdeen AB25 2ZN, United Kingdom
Novartis Investigative Site
Cambridge CB2 0QQ, United Kingdom
Novartis Investigative Site
Cardiff CF14 4XW, United Kingdom
Novartis Investigative Site
Glasgow EH9 1LF, United Kingdom
Novartis Investigative Site
Leeds LS1 3EX, United Kingdom
University College Hospital
London NW1 2BU, United Kingdom
Novartis Investigative Site
London SW17 0QT, United Kingdom
Novartis Investigative Site
Oxford OX3 9DU, United Kingdom
Southampton General Hospital
Southampton SO16 6YD, United Kingdom
How to Get in Touch
Novartis Pharmaceuticals
Sponsor contactCONTACT
Novartis Pharmaceuticals
Sponsor contactCONTACT
