S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)
Status:
Recruiting
Trial end date:
2021-12-31
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1
Details
Lead Sponsor:
Armgo Pharma, Inc. National Institute of Neurological Disorders and Stroke (NINDS)
Collaborators:
Celerion National Institute of Neurological Disorders and Stroke (NINDS) National Institute of Nursing Research (NINR)