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S 48168 (ARM 210) for the Treatment of RYR1-related Myopathies (RYR1-RM)

NCT04141670 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2024-08-22

Study results available
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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.

Conditions

  • RYR-1 Myopathy

Interventions

DRUG

S48168

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

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • National Institute of Nursing Research (NINR)

    collaborator NIH

  • Celerion

    collaborator INDUSTRY

  • Armgo Pharma, Inc.

    lead INDUSTRY

Principal Investigators

  • Payam Mohassel, M.D. · National Institute of Neurological Disorders and Stroke (NINDS)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-08-25
Primary Completion
2022-12-30
Completion
2023-07-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04141670 on ClinicalTrials.gov