A Randomized, Placebo-Controlled, Double-Blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

Study on Investigational Medication for Huntington's Disease, a Brain Condition

C
Christopher James

Primary Investigator

Enrolling By Invitation
21 years - 70 years
All
Phase 3
3 participants needed
1 Location

Brief description of study

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.

THIS STUDY IS ENROLLING BY INVITATION ONLY 

Detailed description of study

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)

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Huntington's Disease
  • Age: 21 years - 70 years
  • Gender: All

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

This study investigates the effects of an investigational medication on people with Huntington's disease, a condition that affects the brain and causes movement, thinking, and emotional problems. The purpose of this study is to see if the investigational medication can slow down the progression of the disease compared to a placebo. A placebo is an inactive substance that looks like the investigational medicine but does not contain any medicine.

Participants in this study will be randomly assigned to receive either the investigational medication or a placebo. The study includes a screening period to check eligibility, a treatment period that lasts up to 36 months, and a follow-up visit. The follow-up visit will happen 30 days after the end of the study, either in person or by phone.

  • Who can participate: Participants must be ambulatory males or females aged 21 to 70 years with a confirmed diagnosis of Huntington's disease. Individuals with a history of gene therapy or certain viral infections are excluded.
  • Study details: Participants will be randomly assigned to receive either the investigational medication or a placebo. They will have regular assessments to monitor their health and the effects of the treatment.
  • Study timelines: The study will last up to 36 months.
Updated on 06 Jul 2026. Study ID: NEUR-NOVARTIS-INVEST-HD, 30320
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only