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

Condition studied

Autosomal Dominant RYR1-Related Myopathy

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from Day 1 in the 1-MSST measurements after approximately 28 days of dosing between active treatment and placebo.

Endpoint detail

1. Change from Day 1 in the 6 MNWT, TUG, 4 SCT, and for weight-scaled muscle strength as measured by QMA and MMT after approximately 28 days of dosing between active treatment and placebo, 2. Change from Day 1 in the PROMIS-F, PROMIS PF and IPAQ questionnaires after approximately 28 days of dosing between active treatment and placebo, 3. Incidence of AEs, serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs) throughout the trial, 4. Change from Day 1 in safety assessments (vital signs, physical examinations, laboratory safety tests, electrocardiograms [ECGs], and Columbia Suicide Severity Rating Scale [C‑SSRS])