Skip to main content
UK clinical trials - updated daily from ClinicalTrials.gov
TrialConnect
← Back to Search
Looking for participantsPhase3

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

Sponsor: Novartis Pharmaceuticals

NCT ID: NCT07326709

View on ClinicalTrials.gov ↗

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)

Huntington Disease

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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

confirmed by positiveincluding a positive

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
Inclusion Criteria: * 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 Criteria: * 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. 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 Other protocol defined inclusion/exclusion criteria may apply

Where Is This Study? (10 UK sites)

Novartis Investigative Site

Exeter EX2 5DW, United Kingdom

Recruiting

Novartis Investigative Site

Aberdeen AB25 2ZN, United Kingdom

Recruiting

Novartis Investigative Site

Cambridge CB2 0QQ, United Kingdom

Recruiting

Novartis Investigative Site

Cardiff CF14 4XW, United Kingdom

Recruiting

Novartis Investigative Site

Glasgow EH9 1LF, United Kingdom

Recruiting

Novartis Investigative Site

Leeds LS1 3EX, United Kingdom

Recruiting

University College Hospital

London NW1 2BU, United Kingdom

Recruiting
Site contact (verified)
Sarah TabriziPrincipal Investigator

Novartis Investigative Site

London SW17 0QT, United Kingdom

Recruiting

Novartis Investigative Site

Oxford OX3 9DU, United Kingdom

Recruiting

Southampton General Hospital

Southampton SO16 6YD, United Kingdom

Recruiting
Site contact (verified)
Christopher KippsPrincipal Investigator

How to Get in Touch

Novartis Pharmaceuticals

Sponsor contact

CONTACT

1-888-669-6682 novartis.email@novartis.com

Novartis Pharmaceuticals

Sponsor contact

CONTACT

+41613241111
Data sourced from ClinicalTrials.gov · Last verified: 2026-09