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A study to evaluate the safety and effectiveness of surlorian in adults with RYR1-related myopathy

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on individuals with Autosomal Dominant RYR1-Related Myopathy, a rare muscle disease caused by specific changes in the RYR1 gene that affect how muscles function. The purpose of the study is to evaluate the effectiveness and safety of a medication called Surlorian, also known as ARM210 or S48168, compared to a placebo.

Participants will receive either the study drug or a placebo in the form of a film-coated tablet taken by oral use. To ensure the results are unbiased, the study uses a double-blind method, meaning neither the participants nor the researchers know which treatment is being administered. Over a period of approximately 28 days, the effects of the medication on muscle strength and how quickly muscles tire will be monitored.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    <b>participation in the trial</b>

    Once enrolled in the study, medication administration begins. surlorian or a placebo (an inactive substance that looks like the real medicine) is assigned.

  2. Step 2

    <b>medication administration</b>

    The assigned medication is taken by oral use (swallowing). if assigned to the surlorian group, the dose is 300 mg in the form of a film-coated tablet.

    This medication period lasts for approximately 28 days.

  3. Step 3

    <b>assessment of muscle strength and function</b>

    After approximately 28 days of taking the medication, several tests are performed to measure changes from the first day. muscle strength and how easily muscles tire are measured using a 1-minute sit-to-stand test (a test where sitting and standing is performed for one minute).

    Additional physical tests include the 6 MNWT, TUG, and 4 SCT to evaluate movement and strength, as well as weight-scaled muscle strength measurements using QMA and MMT.

  4. Step 4

    <b>questionnaires and safety monitoring</b>

    questionnaires such as PROMIS-F, PROMIS PF, and IPAQ are completed to assess physical function and lifestyle.

    safety assessments are conducted throughout the trial, including monitoring vital signs (such as heart rate and blood pressure), physical examinations, laboratory safety tests (blood or urine tests), electrocardiograms (tests that record the electrical activity of the heart), and the Columbia Suicide Severity Rating Scale (a tool used to assess mood and safety).

    Any adverse events (side effects or health changes) are recorded during the entire study period.

Who can join the trial?

4 criteria

  • You must be an adult between the ages of 18 and 65 at the time you agree to join the study.
  • You must have a confirmed genetic diagnosis of Autosomal Dominant RYR1-Related Myopathy, which is a rare muscle disease caused by a specific change in a gene passed down from a parent.
  • You must show signs of weakness in your proximal muscles, which are the muscles located closest to the center of your body, such as those in your hips or shoulders.
  • You must be able to walk 10 meters, either by yourself or by using a cane, but you cannot use any other types of walking assistance.

Who cannot join the trial?

9 criteria

  • Having severe pulmonary dysfunction, which means the lungs are not working well, or experiencing a pulmonary exacerbation, which is a sudden worsening of breathing problems.
  • Having a cardiac disease (heart disease) that a doctor believes could make it harder to measure how well the treatment is working, such as a low left ventricular ejection fraction, which is a measurement of how much blood the heart pumps out with each beat.
  • Having a history of seizure disorders (conditions that cause sudden changes in brain activity), neurologic disease (problems with the brain or nerves), or any neuromuscular disease (conditions affecting the nerves and muscles) other than the specific condition being studied.
  • Having long-term orthopaedic issues (bone or joint problems), any recent injury, or having a planned surgery during the study that might make it difficult to complete the tests.
  • Having high levels of certain liver enzymes, specifically alanine aminotransferase (ALT) or aspartate aminotransferase (AST), which are proteins found in the liver that can indicate liver irritation.
  • Taking certain medications, such as statins (used for cholesterol), proton pump inhibitors, or H2 blockers (used for acid reflux), within a specific timeframe before starting the study medicine.
  • Taking medications that are CYP3A4 substrates, which are substances that the body breaks down using a specific enzyme, within a specific timeframe before the study.
  • Taking medications that are CYP2C8 inhibitors or inducers, which are drugs that either slow down or speed up how a specific enzyme works in the body, within a specific timeframe before the study.
  • Having reported suicidal ideation (thoughts of self-harm) at specific high levels within the last 6 months or having any suicidal behaviour within the last 2 years.
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Investigated drugs

Surlorian is a medication taken by mouth as a tablet. This study is testing how this medicine affects muscle strength and how easily muscles get tired in adults with a specific muscle disease caused by a genetic condition.

What is already known about the treatment

  • Surlorian

    This medication is taken by mouth in the form of a film-coated tablet. Currently being studied in clinical trials, it is being investigated for its potential to help adults with a specific type of muscle disease called RYR1-related myopathy. It works at a molecular level by targeting specific proteins in the muscle cells to help improve muscle strength and reduce tiredness. This substance is classified as an investigational drug for treating rare muscle disorders.

  • Surlorian placebo

    This is an inactive substance taken by mouth that looks exactly like the actual medication. It is used in clinical research to provide a baseline for comparison so that scientists can determine if the real drug is truly effective. It contains no active medicine and does not have a specific therapeutic use or molecular mechanism of action.

Investigated diseases

Autosomal Dominant RYR1-Related Myopathy - This is a rare genetic muscle disorder caused by mutations in the RYR1 gene. It primarily affects the skeletal muscles, leading to weakness and reduced muscle function. The condition typically progresses as the muscles become increasingly less capable of performing tasks. Individuals may experience growing difficulty with physical movements and muscle endurance over time.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-522343-18-00Protocol codeCL2-210-02Estimated enrolment48 patientsSponsorRycarma Therapeutics Inc.

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