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 | Not Yet Recruiting Phase 2

• 2 other identifiers: CL2-210-022025-522343-18-00
• Study Type: interventional
• Target: 28
• Locations: 5 countries
• Sites: 9

Brief 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.

Conditions

Autosomal Dominant RYR1-Related Myopathy

Keywords

RYR1-Related Myopathy; Adults; Phase 2; Interventional; Surlorian; ARM210

Outcome Measures

Primary Outcomes (1)

• Change from baseline in the 1-minute sit-to-stand test (1-MSST)

  Day 1 to day 28 [approximately]

Secondary Outcomes (20)

• Change from baseline in the 6-Minute Walk Test (6-MNWT)

  Day 1 to day 28 [approximately]

• Change from baseline in the Timed Up and Go Test (TUG)

  Day 1 to day 28 [approximately]

• Change from baseline in the 4-Stair Climb Test (4-SCT)

  Day 1 to day 28 [approximately]

• Change from baseline Quantitative Muscle Assessment (QMA)

  Day 1 to day 28 [approximately]

• Change from baseline Manual Muscle Testing (MMT)

  Day 1 to day 28 [approximately]

Study Arms (2)

Group A

EXPERIMENTAL

Drug: Surlorian

Group B

PLACEBO COMPARATOR

Other: Placebo

Interventions

Surlorian DRUG

300 mg administered once a day

Also known as: ARM210 (S48168)

Group A

Placebo OTHER

administered once a day

Group B

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• RyCarma Therapeutics, Inc.: lead

Study Sites (9)

AP-HM- Hôpital de La Timone

Marseille, Bouches-du-Rhône, 13385, France

Location

Institut de Myologie - Hôpital de La Pitié-Salpétrière

Paris, 73013, France

Location

Universitätsklinikum Ulm

Ulm, Baden-Wurttemberg, 89081, Germany

Location

Charité - Campus Berlin Buch

Berlin, State of Berlin, 13125, Germany

Location

Radboud Universitair Medisch Centrum

Nijmegen, Gelderland, 6500 HB, Netherlands

Location

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, Barcelona, 8035, Spain

Location

Hospital Universitario de Donostia

San Sebastián, Guipúzcoa, 20014, Spain

Location

The Robert Jones and Agnes Hunt Orthopaedic Hospital

Oswestry, Shropshire, SY10 7AG, United Kingdom

Location

University College Hospital - PPDS

London, WC1N 3BG, United Kingdom

Location

Central Study Contacts

Patrick Round

CONTACT

888-209-5458; clinicaltrials@rycarma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 7, 2026
• First Posted: April 30, 2026
• Study Start: April 15, 2026
• Primary Completion (Estimated): August 27, 2027
• Study Completion (Estimated): August 27, 2028
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

DE (2); ES (2); FR (2); NL (1); GB (2)