Study aims and goal: A Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP)-SNP3.
No. of study participants and sites: This phase Ia/II b study aims to recruit 36 participants from approximately 25 study sites in Australia, Canada, Denmark, Italy, Germany, Poland, Spain, the UK and the US.
For further information see the clinical trial registration.