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A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Sponsor: Novartis Pharmaceuticals
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.
Official title: A Randomized, Placebo-controlled, Double-blind Phase 3 Study to Evaluate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
Key Details
Gender
All
Age Range
21 Years - 70 Years
Study Type
INTERVENTIONAL
Enrollment
770
Start Date
2026-03-24
Completion Date
2030-04-30
Last Updated
2026-03-30
Healthy Volunteers
No
Conditions
Interventions
Votoplam (blinded)
Votoplam (blinded) active treatment
Placebo
Placebo
Locations (2)
Georgetown University
Washington D.C., District of Columbia, United States
Novartis Investigative Site
Montreal, Quebec, Canada