Adults 18 to 65, any sex, with RYR-1 Myopathy. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants Experiencing Adverse Events When Treated With S48168 (ARM210)Primary· 42 days
Composite safety and tolerability profile of S48168 (ARM210) based on adverse event reporting
Participants with fatal adverse events (all cause mortality)
Group
Value
95% CI
Low Dose Group
0
High Dose Group
0
Participants with serious adverse events
Group
Value
95% CI
Low Dose Group
0
High Dose Group
0
Participants with non-serious adverse events
Group
Value
95% CI
Low Dose Group
3
High Dose Group
3
Adverse events — posted to ClinicalTrials.gov
Time frame: Approximately two months.
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This study proposes to test S 48168 (ARM210) in a Phase 1 trial in RYR1-RM patients, specifically. The objectives of this study are to explore the safety and tolerability, pharmacokinetics (PK), pharmacodynamics (PD)/target engagement (TE) of S 48168 (ARM210), as well as effects on muscle/motor function, and fatigue in RYR1-RM patients. The study population will include adult patients (≥18 years of age) who have demonstrated leaky RyR1 channels that are responsive to S48168 (ARM210) ex vivo.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by RyCarma Therapeutics, Inc.
Last refreshed: 22 August 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04141670.