Phase2 Trial - Surlorian compared to Placebo in Adults with Autosomal Dominant RYR1-Related Myopathy
Research type
Research Study
Full title
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
IRAS ID
1012795
Contact name
Sasha Barrate
Contact email
Sponsor organisation
RyCarma Therapeutics, Inc.
Research summary
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial in adults with autosomal dominant ryanodine receptor 1-related myopathy (RYR1-RM) with an optional open label extension (OLE) phase. RYR1-RM is a rare muscle disease caused by changes in the RYR1 gene.
People with RYR1-RM may be slower to walk, stand; have trouble walking and climbing stairs; and have muscle weakness. In serious cases, they might also have a curved spine (scoliosis), problems moving their eyes, and trouble breathing because their muscles are weak.
This study will test surlorian, a new investigational drug being developed to treat RYR1-RM. The goals of this study are to see how well surlorian works to improve muscle strength and decrease muscle fatigue in participants with RYR1-RM. The study is also planned to see if surlorian is safe and tolerable in study participants.
The planned duration of the trial is approximately 16 weeks. The trial consists of Screening (<28 days), Treatment (2 periods of 28 days each), Washout (21 days ± 7 days) and Follow-up (7 days after each period). The planned duration for the OLE phase will be up to 12 months for participants who enrol into this phase.
During the treatment period, participants will either receive study drug (either surlorian or placebo) and have equal chances of taking either of them first. There are 2 Treatment Periods each 28-days long. In the second Treatment Period, participants will take the study drug that they did not take during the first Treatment Period. All participants who enrol in the OLE phase will receive surlorian. During each 28-day Treatment Period and OLE phase, participants will take 3 tablets of the study drug once daily.
About 28 adults will take part in this worldwide study. This study will be run at about 9 sites in different countries.REC name
London - Chelsea Research Ethics Committee
REC reference
25/LO/0909
Date of REC Opinion
4 Feb 2026
REC opinion
Further Information Favourable Opinion