NCT07326709

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

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 770 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 21 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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)

Conditions

• Huntington Disease

Interventions

Type	Name	Description
DRUG	Votoplam (blinded)	Votoplam (blinded) active treatment
DRUG	Placebo	Placebo

Timeline

• Start date: 2026-03-24
• Primary completion: 2030-04-02
• Completion: 2030-04-30
• First posted: 2026-01-08
• Last updated: 2026-04-16

Locations

4 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study