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A trial to compare treatment with surlorian (ARM210, S48168) to placebo in effects on muscle strength and safety in adults with autosomal dominant RYR1-related myopathy
EU CTIS ID: 2025-522343-18-00
What this study is testing
To determine the effect of surlorian on muscle strength and fatigability using 1-minute sit-to-stand test (1-MSST)
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Is an adult aged 18-65 years at the time of signing informed consent.
- 2. Has a confirmed genetic diagnosis of RYR1-RM with autosomal dominant mutation.
- 3. Has clinical evidence of weakness affecting any proximal muscle group(s) as assessed by the Investigator.
- 4. Can walk 10 m with or without a cane (no other walking aid allowed).
You likely can't join if
- 1. Has severe pulmonary dysfunction at screening (e.g., FVC <40% predicted) or evidence of pulmonary exacerbation. Pulmonary exacerbation is an acute worsening of respiratory symptoms that result from a decline in lung function.
- 2.Has cardiac disease by history or at screening that in Investigator’s judgment is likely to worsen overall performance on efficacy measures during the trial (e.g., left ventricular ejection fraction < 40%).
- 3. Has a history of seizure disorder, neurologic disease, or neuromuscular disease other than RYR1-RM.
- 4. Has a history of chronic orthopaedic issues, any acute injury or expected surgery during the trial that may affect the ability to complete trial assessments.
- 5. Participants with screening alanine aminotransferase (ALT) levels >3 × upper limit of normal (ULN) or screening aspartate aminotransferase (AST) levels >5 × ULN (isolated elevations of total bilirubin <2 × ULN with direct bilirubin below the ULN will be included).
- 6. Received treatment with statins, proton pump inhibitors, or H2 blockers within 7 days or 5 half-lives, whichever is longer, prior to the first dose of IP.
See the full eligibility criteria
- 1. Is an adult aged 18-65 years at the time of signing informed consent.
- 2. Has a confirmed genetic diagnosis of RYR1-RM with autosomal dominant mutation.
- 3. Has clinical evidence of weakness affecting any proximal muscle group(s) as assessed by the Investigator.
- 4. Can walk 10 m with or without a cane (no other walking aid allowed).
- 1. Has severe pulmonary dysfunction at screening (e.g., FVC <40% predicted) or evidence of pulmonary exacerbation. Pulmonary exacerbation is an acute worsening of respiratory symptoms that result from a decline in lung function.
- 2.Has cardiac disease by history or at screening that in Investigator’s judgment is likely to worsen overall performance on efficacy measures during the trial (e.g., left ventricular ejection fraction < 40%).
- 3. Has a history of seizure disorder, neurologic disease, or neuromuscular disease other than RYR1-RM.
- 4. Has a history of chronic orthopaedic issues, any acute injury or expected surgery during the trial that may affect the ability to complete trial assessments.
- 5. Participants with screening alanine aminotransferase (ALT) levels >3 × upper limit of normal (ULN) or screening aspartate aminotransferase (AST) levels >5 × ULN (isolated elevations of total bilirubin <2 × ULN with direct bilirubin below the ULN will be included).
- 6. Received treatment with statins, proton pump inhibitors, or H2 blockers within 7 days or 5 half-lives, whichever is longer, prior to the first dose of IP.
- 7. Received treatment with sensitive or narrow therapeutic index CYP3A4 substrates within 7 days or 5 half-lives, whichever is longer, prior to the first dose of IP.
- 8. Received treatment with strong or moderate CYP2C8 inhibitors or inducers within 7 days or 5 half-lives, whichever is longer, prior to the first dose of IP.
- 9. Has reported any suicidal ideation of Category 4 or 5 on the C-SSRS within 6 months prior to screening or any suicidal behaviour in the last 2 years prior to screening, as indicated by any ‘yes’ answers on the suicidal behaviour section of C-SSRS.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.