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

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What is this study about?

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.

Who Can Join the Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name City Country Status
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Stichting Radboud University Medical Center Nijmegen The Netherlands
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Hopital Beaujon Clichy France
Alyjslpkqd Pejzdgcp Hhbzqavg Dl Munyqxrhr Marseille France
Hsfagwny Uxwxxpufwmdtx Dkpnixdb Donostia / San Sebastian Spain
Hiyafjhf Vffw dupvldcd Barcelona Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Not yet recruiting
30.04.2026
Germany Germany
Not yet recruiting
30.04.2026
Spain Spain
Not yet recruiting
30.04.2026
The Netherlands The Netherlands
Not yet recruiting
30.04.2026

Trial locations

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.

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 ID:
2025-522343-18-00
Protocol code:
CL2-210-02
Trial Phase:
Therapeutic exploratory (Phase II)

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