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Clinical Trials in the UK / NCT07560020
Recruiting Phase 2

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

NCT07560020 · tracked via the Priya Life Science UK tracker
Sponsor
RyCarma Therapeutics, Inc.
Phase
Phase 2
Started
2026-07-01
Last updated
2026-07-15

Condition(s) studied

Autosomal Dominant RYR1-Related Myopathy

Investigational drug(s) / intervention(s)

SurlorianPlacebo

Surlorian: 300 mg administered once a day

Placebo: administered once a day

Study summary

This study is testing a medicine called surlorian in adults who have a genetic muscle condition known as autosomal dominant RYR1-related myopathy (RYR1-RM). The goal is to find out whether surlorian improves muscle weakness, and whether it is safe and well tolerated.

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria: * Can understand the written informed consent, provides signed and witnessed written informed consent, and agrees to comply with all protocol requirements * Confirmed genetic diagnosis of RYR1-RM with autosomal dominant mutation * Clinical evidence of weakness affecting any proximal muscle group(s) as assessed by the Investigator * Can walk 10 m with or without a cane (no other walking aid allowed) * Is either a female of non-childbearing potential or male or female, who agrees to use highly effective contraception/preventive exposure measures from the time of first dose of IP (for a male participant) or the signing of the informed consent form (ICF) (for a female participant) during the trial, and until 7 days after the last dose of IP. Exclusion Criteria: * Unable or unwilling to understand and comply with protocol requirements or unlikely to complete the study as planned, as judged by the Investigator * Any clinically significant medical condition that, in the opinion of the Investigator, would interfere with the study * Females who are pregnant, breastfeeding or intend to become pregnant, or of childbearing potential not using adequate contraceptive methods * Participants with severe pulmonary dysfunction at screening, or evidence of pulmonary exacerbation (defined as an acute worsening of respiratory symptoms that result from a decline in lung function) * Cardiac disease by history or at screening that, in the Investigator's opinion, is likely to worsen overall performance of efficacy measures during the study * History of seizure disorder, neurologic disease, or neuromuscular disease other than RYR1-RM * History of chronic orthopedic issues, acute injury, or expected surgery during the study that may affect the ability to complete study assessments * Positive test results at screening for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV) * 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) * History within the past year of alcohol or other drug substance abuse * Known hypersensitivity to the investigational product of related compounds * 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 the study drug * Treatment with sensitive or narrow therapeutic index CYP3A4 substrates within 7 days or 5 half-lives, whichever is longer, prior to first dose of the study drug * Treatment with strong or moderate CYP2C8 inhibitor or inducers within 7 days or 5 half-lives, whichever is longer, prior to the first dose of the study drug * Currently enrolled in another study or received treatment with any other investigational drug within 30 days or \> 5 half-lives, whichever is longer, prior to screening * Has reported any suicide ideation of Category 4 or 5 on the C-SSRS within 6 months prior to screening or any suicidal behavior in the last two years prior to screening as indicated by any 'yes' answers on the suicidal behavior section of the C-SSRS

Primary outcome measure(s)

Trial sites (9)

FacilityCityRegionStatus
AP-HM- Hôpital de La Timone Marseille Bouches-du-Rhône Not Yet Recruiting
Institut de Myologie - Hôpital de La Pitié-Salpétrière Paris France Not Yet Recruiting
Universitätsklinikum Ulm Ulm Baden-Wurttemberg Not Yet Recruiting
Charité - Campus Berlin Buch Berlin State of Berlin Recruiting
Radboud Universitair Medisch Centrum Nijmegen Gelderland Not Yet Recruiting
Hospital Universitario Vall d'Hebron - PPDS Barcelona Barcelona Not Yet Recruiting
Hospital Universitario de Donostia San Sebastián Guipúzcoa Not Yet Recruiting
The Robert Jones and Agnes Hunt Orthopaedic Hospital Oswestry Shropshire Not Yet Recruiting
University College Hospital - PPDS London United Kingdom Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07560020 on ClinicalTrials.gov ↗ ← All trials in the UK