CI

At a glance

ClinicalIndex Comparison Record
Phase 1Completed· 7 enrolled
Drug / intervention
S48168drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04141670
NCT04141670Phase 1Completed
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Safety and Tolerability of S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)

RyCarma Therapeutics, Inc.·interventional·Posted Oct 28, 2019·Updated Aug 22, 2024

In Brief

A Phase 1 clinical trial evaluating S48168 for RYR-1 Myopathy. Completed, enrolled 7 participants across 1 site.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsRYR-1 Myopathy
CountriesUnited States
CollaboratorsNational Institute of Neurological Disorders and Stroke (NINDS), National Institute of Nursing Research (NINR), Celerion

Timeline

Phase 1CompletedFinished
2020202120222023202420252026
First PostedOct 28, 2019
Enrollment StartAug 25, 2020
Primary CompletionDec 30, 2022
Study CompletionJul 30, 2023
TodayJul 2, 2026
Enrollment to primary: 2.3 yearsPosted 6.7 years ago

Interventions

S48168drug

A novel oral small molecule which is designed to repair leaky RYR1 channels