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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
2026-05-27
Healthy Volunteers
No
Conditions
Interventions
Votoplam (blinded)
Votoplam (blinded) active treatment
Placebo
Placebo
Locations (13)
CenExcel Rocky Mtn Clin Research
Englewood, Colorado, United States
Georgetown University
Washington D.C., District of Columbia, United States
Albany Medical College
Albany, New York, United States
UBMD Neurology
Buffalo, New York, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
TX Movement Disorder Spec PLLC
Georgetown, Texas, United States
U of TX Health Science Ct
Houston, Texas, United States
University of Washington Medical Center
Seattle, Washington, United States
North York General Hospital
North York, Ontario, Canada
Centre de recherche du CHUM
Montreal, Quebec, Canada
CUSM Montreal Neurological Institute
Montreal, Quebec, Canada
Novartis Investigative Site
London, United Kingdom
Novartis Investigative Site
Southampton, United Kingdom