Phase 2 Trial Comparing Surlorian and Placebo for Muscle Weakness in Adults With Autosomal Dominant RYR1-Related Myopathy

What this Trial Is About

Researchers are evaluating the medicine surlorian in adults with autosomal dominant RYR1-related myopathy RYR1-RM, a genetic muscle condition. The study aims to find out if surlorian improves muscle weakness and to assess its safety and tolerability. This phase 2 trial involves adults aged 18 to 65 years who have confirmed genetic diagnosis and muscle weakness affecting proximal muscles. Participants will take part in a double-blind, randomized crossover study where they receive surlorian or a placebo once daily in two treatment periods separated by a washout. Each treatment period lasts about 16 weeks. After completing the main study, participants may join an optional open-label extension of about 12 months where everyone receives surlorian. During the study, participants undergo muscle strength and physical function tests like the 1-minute sit-to-stand test and 6-minute walk test. Researchers will also monitor blood pressure, blood tests, and adverse events for safety. The total study duration is approximately 68 weeks, including treatment and follow-up assessments to measure changes from baseline and evaluate tolerability.

A Trial to Compare Treatment With Surlorian (ARM210, S48168) to Placebo in Effects on Muscle Strength and Safety in Adults With Autosomal Dominant RYR1-related Myopathy