Autosomal Dominant RYR1-Related MyopathyPhase 2NCT07560020
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
A Phase 2 study testing Surlorian in people with Autosomal Dominant RYR1-Related Myopathy, run by RyCarma Therapeutics, Inc. at 9 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Autosomal Dominant RYR1-Related Myopathy
- What is being tested?
- Surlorian
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 28 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 9 Sep 2026to 27 Aug 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
9 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
9 sites in 5 countries
France2 sites, 2 recruiting
- Not yet recruiting
AP-HM- Hôpital de La Timone
Marseille, Bouches-du-Rhône, 13385
- Recruiting
Institut de Myologie - Hôpital de La Pitié-Salpétrière
Paris, 73013
Professor Ana Ferreiro PU-PH, essais-adultes@institut-myologie.org
Ana Ferreiro, PU-PH
Germany2 sites, 2 recruiting
- Recruiting
Charité - Campus Berlin Buch
Berlin, State of Berlin, 13125
+ 49 30 450 540 507, myologie@charite.de
Simone Spuler, MD
- Not yet recruiting
Universitätsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081
Nam Nguyen-Younossi, MD, nam.nguyen-younossi@uniklinik-ulm.de
Angela Rosenbohm, MD
Spain2 sites, 2 recruiting
- Not yet recruiting
Hospital Universitario Vall d'Hebron - PPDS
Barcelona, Barcelona, 8035
- Not yet recruiting
Hospital Universitario de Donostia
San Sebastián, Guipúzcoa, 20014
United Kingdom2 sites, 2 recruiting
- Recruiting
University College Hospital - PPDS
London, WC1N 3BG
Laura Berg, Laura.berg1@nhs.net
Chiara Pizzamiglio, MD PhD
- Recruiting
The Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, Shropshire, SY10 7AG
Site Clinical Research Contact, +44 1691 404210 (or 404142), rjah.researchoffice@nhs.net
Natalie Grigg, natalie.grigg4@nhs.net
Netherlands1 site, 1 recruiting
- Not yet recruiting
Radboud Universitair Medisch Centrum
Nijmegen, Gelderland, 6500 HB
Spierziekten Research Team Radboudumc, spierziektenresearchteam@radboudumc.nl
Nicol Voermans, MD PhD
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Efficacy and Safety of Surlorian (ARM210, S48168) in Adults With Autosomal Dominant RYR1-Related Myopathy
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.
Read the detailed description
The study is taking place at several medical centers with doctors who specialize in treating people with RYR1-RM. Everyone in the study will receive both surlorian and a placebo (a "dummy" treatment) at different times, but neither the participants nor the study staff will know which one they are getting during each period.
During Treatment period 1, participants will be randomly assigned to receive either surlorian or a placebo, which will be followed up by a washout period. Following the washout, in Treatment period 2, participants will switch and receive the opposite treatment from what they received in the first period. This main part of the study lasts about 16 weeks.
After finishing the main, placebo-controlled part of the study participants may be able to join an open-label extension lasting approximately 12 months. In this extension, everyone receives surlorian.
- Conditions
- Autosomal Dominant RYR1-Related Myopathy
- Being tested
- Surlorian, Placebo
- Keywords
- RYR1-Related Myopathy, Adults, Phase 2, Interventional, Surlorian, ARM210
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SurlorianDrug
Also called ARM210 (S48168)
300 mg administered once a day
PlaceboOther
administered once a day
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | Not described Receives: Drug: Surlorian |
| Group B | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in the 1-minute sit-to-stand test (1-MSST)
Measured: Day 1 to day 28 [approximately]
Secondary outcomes (20)
Change from baseline in the 6-Minute Walk Test (6-MNWT)
Measured: Day 1 to day 28 [approximately]
Change from baseline in the Timed Up and Go Test (TUG)
Measured: Day 1 to day 28 [approximately]
Change from baseline in the 4-Stair Climb Test (4-SCT)
Measured: Day 1 to day 28 [approximately]
Change from baseline Quantitative Muscle Assessment (QMA)
Measured: Day 1 to day 28 [approximately]
Change from baseline Manual Muscle Testing (MMT)
Measured: Day 1 to day 28 [approximately]
Change from baseline in Patient-Reported Outcomes Measurement Information System-fatigue (PROMIS-F)
Measured: Day 1 to day 28 [approximately]
Change from baseline in Patient-Reported Outcomes Measurement Information System-physical fatigue (PROMIS-PF)
Measured: Day 1 to day 28 [approximately]
Chage in International Physical Activity Questionnaire (IPAQ)
Measured: Day 1 to day 28 [approximately]
Number of Adverse Events
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in systolic blood pressure
Systolic blood pressure will be measured in millimeters of mercury (mmHg)
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in diastolic blood pressure
Diastolic blood pressure will be measured in millimeters of mercury (mmHg)
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in temperature
Temperature will be measured in degrees Centigrade (°C)
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in hemoglobin
Hemoglobin concentration will be measured in grams per liter (g/L) using standard hematology laboratory methods
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in white blood cell count
White blood cell count will be measured in ×10⁹/L using standard hematology laboratory methods
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in platelet count
Platelet count will be measured in ×10⁹/L using standard hematology laboratory methods
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in serum creatinine
Serum creatinine will be measured in micromoles per liter (µmol/L) using standard clinical chemistry methods
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in estimated glomerular filtration rate
Estimated glomerular filtration rate (eGFR) will be calculated from serum creatinine using the Chronic Kidney Disease Epidemiology Collaboration equation
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in serum glucose
Serum glucose concentration will be measured in millimoles per liter (mmol/L) using standard clinical chemistry methods
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in QTc interval
Corrected QT interval (QTcF) will be measured in milliseconds using triplicate 12-lead electrocardiograms
Measured: Day 1 to end of study [approximately 68 weeks]
Change from baseline in urine protein
Urine protein will be measured in milligrams per liter (mg/L) using standard urinalysis methods
Measured: Day 1 to end of study [approximately 68 weeks]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2026
- Study start9 Sep 2026
- Primary completion27 Aug 2027 (estimated)
- Study completion27 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autosomal Dominant RYR1-Related MyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- RyCarma Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07560020
- Sponsor's protocol ID
- CL2-210-02
- Other IDs
- 2025-522343-18-00, 2025
- Status last verified
- Sep 2026
- First submitted
- 7 Apr 2026
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No