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