A randomized, placebo-controlled, double-blind Phase 3 study to evaluate the efficacy, safety and tolerability of votoplam in participants with Huntington’s Disease

Condition studied

Huntington’s Disease

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from Baseline to Month 36 in cUHDRS score

Endpoint detail

Change from Baseline to Month 36 in UHDRS-TFC, Change from Baseline to Month 36 in UHDRS-IS, The time to decline in TFC score by at least one or IS score by at least 10 over the treatment period, Change from Baseline to month 36 in UHDRS-TMS, Change from Baseline to Month 36 in SDMT, Change from Baseline to Month 36 in SWRT, Percent change from Baseline to steady state in blood mHTT protein, Change from Baseline to Month 36 in serum NfL, Incidence and severity of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and TEAEs leading to participant withdrawal