Phase 2 Study of Oral VIM0423 for Treating Isolated Dystonia Affecting Multiple Body Parts Evaluating Safety, Effectiveness, and Tolerability Compared to Placebo

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of VIM0423, an oral medication, in adults with isolated dystonia affecting two or more body regions. This Phase 2, randomized, double-blind, placebo-controlled study aims to determine whether VIM0423 improves dystonia symptoms compared to placebo and whether it enhances daily function and quality of life. The trial addresses a significant need as many individuals lack approved treatments beyond botulinum toxin injections for limited dystonia types. Participants will be randomly assigned to receive either VIM0423 or a matching placebo, with up to six pills taken nightly before bed for 16 weeks. The study includes several phases screening and baseline period lasting up to 14 weeks with two visits, a 16-week treatment phase consisting of dose titration, maintenance, and taper with three visits, followed by a 2-week safety follow-up visit. The trial enrolls up to 120 adults aged 18 to 65 with isolated dystonia. Throughout participation, individuals will attend six in-person visits during the trial lasting up to 32 weeks. Assessments include clinical evaluations, video recordings to monitor dystonia changes, laboratory tests, EKGs, and monitoring for adverse events. Participants will also self-assess dystonia impact on daily living. The main outcome measured is the change in the Burke-Fahn-Marsden Dystonia Rating Scale from baseline to week 14, alongside other clinical and patient global impression scales to evaluate symptom severity and change.

A Study to Evaluate the Efficacy, Safety, and Tolerability of VIM0423 in Individuals With Isolated Dystonia