Phase 3 Study to Evaluate How Well Votoplam Works and Its Safety in Adults With Huntingtons Disease

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of votoplam in slowing disease progression in patients with early symptomatic Huntingtons disease. This Phase 3 study compares votoplam to a placebo control group to better understand its impact on this genetic condition. The study is sponsored by Novartis Pharmaceuticals and uses the compound HTT227 formerly PTC518. Participants will be randomly assigned in a 32 ratio to receive either votoplam or placebo, both taken orally. The trial has three periods a screening period of up to 42 days to determine eligibility a double-blind treatment period lasting up to 36 months, with variable individual treatment durations and a safety follow-up period consisting of one visit or phone call 30 days after the end of the study for participants not continuing in an open-label extension. During the study, participants will undergo regular assessments including measurements of neurological function through the cUHDRS score and other clinical scales at baseline and month 36. Blood tests will monitor levels of mutant huntingtin protein and serum neurofilament light. Safety will be evaluated by tracking adverse events throughout treatment. This comprehensive follow-up aims to gather detailed information on disease progression and treatment effects over up to three years.

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