SURLORIAN for Autosomal dominant RYR1-related myopathy | Congenital myopathy

Inclusion criteria

• {"criterion_text":"- 1. Is an adult aged 18-65 years at the time of signing informed consent.\n- 2. Has a confirmed genetic diagnosis of RYR1-RM with autosomal dominant mutation.\n- 3. Has clinical evidence of weakness affecting any proximal muscle group(s) as assessed by the Investigator.\n- 4. Can walk 10 m with or without a cane (no other walking aid allowed)."}

Exclusion criteria

• {"criterion_text":"- 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.\n- 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%).\n- 3. Has a history of seizure disorder, neurologic disease, or neuromuscular disease other than RYR1-RM.\n- 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.\n- 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).\n- 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.\n- 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.\n- 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.\n- 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."}

Primary endpoints

• {"endpoint_text":"- Change from Day 1 in the 1-MSST measurements after approximately 28 days of dosing between active treatment and placebo.","definition_or_measurement_approach":"1-minute sit-to-stand test (1‑MSST); change from Day 1 measured after ~28 days of dosing comparing active treatment and placebo."}

Secondary endpoints

• {"endpoint_text":"- 1. Change from Day 1 in the 6 MNWT, TUG, 4 SCT, and for weight-scaled muscle strength as measured by QMA and MMT after approximately 28 days of dosing between active treatment and placebo","definition_or_measurement_approach":"6-Minute Walk Test (6-MNWT), Timed Up and Go (TUG), 4-Stair Climb Test (4-SCT), Quantitative Muscle Assessment (QMA) and Manual Muscle Testing (MMT); change from Day 1 after ~28 days dosing comparing active vs placebo."}
• {"endpoint_text":"- 2. Change from Day 1 in the PROMIS-F, PROMIS PF and IPAQ questionnaires after approximately 28 days of dosing between active treatment and placebo","definition_or_measurement_approach":"Patient-reported outcome measures: PROMIS-F (fatigue), PROMIS-PF (physical function) and IPAQ; change from Day 1 after ~28 days dosing comparing active vs placebo."}
• {"endpoint_text":"- 3. Incidence of AEs, serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs) throughout the trial","definition_or_measurement_approach":"Safety surveillance throughout the trial capturing AEs, SAEs, TEAEs and AESIs as reported during study visits and follow-up."}
• {"endpoint_text":"- 4. Change from Day 1 in safety assessments (vital signs, physical examinations, laboratory safety tests, electrocardiograms [ECGs], and Columbia Suicide Severity Rating Scale [C‑SSRS])","definition_or_measurement_approach":"Safety assessments including vital signs, physical exams, laboratory safety tests, ECGs, and C-SSRS; change from baseline (Day 1)."}

Spain

Earliest CTIS Part Ii Submission Date

25-03-2026

Latest Decision Or Authorization Date

07-04-2026

Processing Time Days

13

Number Of Sites

2

Number Of Participants

7

France

Earliest CTIS Part Ii Submission Date

24-03-2026

Latest Decision Or Authorization Date

07-04-2026

Processing Time Days

14

Number Of Sites

2

Number Of Participants

7

Germany

Earliest CTIS Part Ii Submission Date

13-03-2026

Latest Decision Or Authorization Date

02-04-2026

Processing Time Days

20

Number Of Sites

2

Number Of Participants

21

Netherlands

Earliest CTIS Part Ii Submission Date

16-03-2026

Latest Decision Or Authorization Date

02-04-2026

Processing Time Days

17

Number Of Sites

1

Number Of Participants

6

Primary sponsor

Full Name

Rycarma Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

PPD Development LP

Responsibilities

Extensive study support functions (sponsorDuties codes: [1,10,11,12,13,2,5,6,8])

Name

PPD Global Central Labs

Responsibilities

Central laboratory services (sponsorDuties code: [4])

Name

Fisher Clinical Services GmbH

Responsibilities

Supply/logistics (sponsorDuties code: [14])

Name

Suvoda LLC

Responsibilities

eClinical/IRT or data tools (sponsorDuties code: [3])

Name

Medidata Solutions Inc.

Responsibilities

eClinical platform services (sponsorDuties code: [7])

Third parties

• {"country":"France","full_name":"Sysnav","duties_or_roles":"sponsorDuties codes: [7]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: [7]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Clinical Trial Participant Travel, Payment, and Expense Reimbursement Management (code 15)","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"sponsorDuties codes: [3]","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"sponsorDuties codes: [14]","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: [1,10,11,12,13,2,5,6,8]","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Fisher Clinical Services GmbH (Weil Am Rhein site)","duties_or_roles":"sponsorDuties codes: [14]","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"FGK Representative Service B.V.","duties_or_roles":"Legal Representation (code 15)","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Nuvisan GmbH","duties_or_roles":"sponsorDuties codes: [4]","organisation_type":"Pharmaceutical company"}