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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Rycarma Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Decision date (initial)

2026-04-07

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

RyCarma Therapeutics, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacokinetic, Safety

To determine the effect of surlorian on muscle strength and fatigability using 1-minute sit-to-stand test (1-MSST)

Secondary objectives 3

1. • To determine the effect of surlorian on muscle strength and fatigability using the 6-Minute Walk Test (6-MNWT), Timed Up and Go Test (TUG), 4-Stair Climb Test (4-SCT), Quantitative Muscle Assessment (QMA), and Manual Muscle Testing (MMT)
2. • To determine the effect of surlorian on participant-reported fatigue and physical function
3. • To determine the safety and tolerability of surlorian

Conditions and MedDRA coding

Autosomal Dominant RYR1-Related Myopathy

Study design 7 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

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

Exclusion criteria 9

1. 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. 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. 3. Has a history of seizure disorder, neurologic disease, or neuromuscular disease other than RYR1-RM.
4. 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. 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. 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. 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. 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. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from Day 1 in the 1-MSST measurements after approximately 28 days of dosing between active treatment and placebo.

Secondary endpoints 4

1. 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
2. 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
3. 3. Incidence of AEs, serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs) throughout the trial
4. 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])

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Rycarma Therapeutics Inc.

Sponsor organisation

Rycarma Therapeutics Inc.

Address

200 Clarendon Street

City

Boston

Postcode

02116-5021

Country

United States

Scientific contact point

Organisation

Rycarma Therapeutics Inc.

Contact name

Elizabeth Tarka

Public contact point

Organisation

Rycarma Therapeutics Inc.

Contact name

Sanjay Jalota

Third parties 10

Locations

4 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 48 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications