Phase 2Recruiting
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
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Efficacy and Safety of Surlorian (ARM210, S48168) in Adults With Autosomal Dominant RYR1-Related Myopathy
- Sponsor
- RyCarma Therapeutics, Inc. (Industry)
- Enrolment
- 28 planned
- Conditions
- Autosomal Dominant RYR1-Related Myopathy
- Interventions
- Surlorian; Placebo
- Ages
- adults, older adults
- Registry
- NCT07560020
Full record on ClinicalTrials.gov
From the registry
This study is testing a medicine called surlorian in adults who have a genetic muscle condition known as autosomal dominant RYR1-related myopathy (RYR1-RM). The goal is to find out whether surlorian improves muscle weakness, and whether it is safe and well tolerated.
Primary outcomes
- Change from baseline in the 1-minute sit-to-stand test (1-MSST)
History
Study completion expected
Primary completion expected
Last registry update
Started
Registered