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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

13 juli 2026 uppdaterad av: RyCarma Therapeutics, Inc.

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.

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Detaljerad beskrivning

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.

Studietyp

Interventionell

Inskrivning (Beräknad)

28

Fas

  • Fas 2

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

  • Frankrike
      • Paris, Frankrike, 73013
        • Har inte rekryterat ännu
        • Institut de Myologie - Hôpital de La Pitié-Salpétrière
    • Bouches-du-Rhône
      • Marseille, Bouches-du-Rhône, Frankrike, 13385
        • Har inte rekryterat ännu
        • AP-HM- Hôpital de La Timone
  • Nederländerna
    • Gelderland
      • Nijmegen, Gelderland, Nederländerna, 6500 HB
        • Har inte rekryterat ännu
        • Radboud Universitair Medisch Centrum
  • Spanien
    • Barcelona
      • Barcelona, Barcelona, Spanien, 8035
        • Har inte rekryterat ännu
        • Hospital Universitario Vall d'Hebron - PPDS
    • Guipúzcoa
      • San Sebastián, Guipúzcoa, Spanien, 20014
        • Har inte rekryterat ännu
        • Hospital Universitario de Donostia
  • Storbritannien
      • London, Storbritannien, WC1N 3BG
        • Rekrytering
        • University College Hospital - PPDS
    • Shropshire
      • Oswestry, Shropshire, Storbritannien, SY10 7AG
        • Har inte rekryterat ännu
        • The Robert Jones and Agnes Hunt Orthopaedic Hospital
  • Tyskland
    • Baden-Wurttemberg
      • Ulm, Baden-Wurttemberg, Tyskland, 89081
        • Har inte rekryterat ännu
        • Universitätsklinikum Ulm
    • State of Berlin
      • Berlin, State of Berlin, Tyskland, 13125
        • Rekrytering
        • Charité - Campus Berlin Buch

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Beskrivning

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

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Crossover tilldelning
  • Maskning: Dubbel

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Grupp A
Läkemedel: Surlorian
300 mg administered once a day
Andra namn:
  • ARM210 (S48168)
Placebo-jämförare: Grupp B
Övrig: Placebo
administered once a day

Vad mäter studien?

Primära resultatmått

Resultatmått
Tidsram
Change from baseline in the 1-minute sit-to-stand test (1-MSST)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Change from baseline in the 6-Minute Walk Test (6-MNWT)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in the Timed Up and Go Test (TUG)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in the 4-Stair Climb Test (4-SCT)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline Quantitative Muscle Assessment (QMA)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline Manual Muscle Testing (MMT)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in Patient-Reported Outcomes Measurement Information System-fatigue (PROMIS-F)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in Patient-Reported Outcomes Measurement Information System-physical fatigue (PROMIS-PF)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Chage in International Physical Activity Questionnaire (IPAQ)
Tidsram: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Number of Adverse Events
Tidsram: Day 1 to end of study [approximately 68 weeks]
Day 1 to end of study [approximately 68 weeks]
Change from baseline in systolic blood pressure
Tidsram: Day 1 to end of study [approximately 68 weeks]
Systolic blood pressure will be measured in millimeters of mercury (mmHg)
Day 1 to end of study [approximately 68 weeks]
Change from baseline in diastolic blood pressure
Tidsram: Day 1 to end of study [approximately 68 weeks]
Diastolic blood pressure will be measured in millimeters of mercury (mmHg)
Day 1 to end of study [approximately 68 weeks]
Change from baseline in temperature
Tidsram: Day 1 to end of study [approximately 68 weeks]
Temperature will be measured in degrees Centigrade (°C)
Day 1 to end of study [approximately 68 weeks]
Change from baseline in hemoglobin
Tidsram: Day 1 to end of study [approximately 68 weeks]
Hemoglobin concentration will be measured in grams per liter (g/L) using standard hematology laboratory methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in white blood cell count
Tidsram: Day 1 to end of study [approximately 68 weeks]
White blood cell count will be measured in ×10⁹/L using standard hematology laboratory methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in platelet count
Tidsram: Day 1 to end of study [approximately 68 weeks]
Platelet count will be measured in ×10⁹/L using standard hematology laboratory methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in serum creatinine
Tidsram: Day 1 to end of study [approximately 68 weeks]
Serum creatinine will be measured in micromoles per liter (µmol/L) using standard clinical chemistry methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in estimated glomerular filtration rate
Tidsram: Day 1 to end of study [approximately 68 weeks]
Estimated glomerular filtration rate (eGFR) will be calculated from serum creatinine using the Chronic Kidney Disease Epidemiology Collaboration equation
Day 1 to end of study [approximately 68 weeks]
Change from baseline in serum glucose
Tidsram: Day 1 to end of study [approximately 68 weeks]
Serum glucose concentration will be measured in millimoles per liter (mmol/L) using standard clinical chemistry methods
Day 1 to end of study [approximately 68 weeks]
Change from baseline in QTc interval
Tidsram: Day 1 to end of study [approximately 68 weeks]
Corrected QT interval (QTcF) will be measured in milliseconds using triplicate 12-lead electrocardiograms
Day 1 to end of study [approximately 68 weeks]
Change from baseline in urine protein
Tidsram: Day 1 to end of study [approximately 68 weeks]
Urine protein will be measured in milligrams per liter (mg/L) using standard urinalysis methods
Day 1 to end of study [approximately 68 weeks]

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

RyCarma Therapeutics, Inc.

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Beräknad)

1 juli 2026

Primärt slutförande (Beräknad)

27 augusti 2027

Avslutad studie (Beräknad)

27 augusti 2028

Studieregistreringsdatum

Först inskickad

7 april 2026

Först inskickad som uppfyllde QC-kriterierna

23 april 2026

Första postat (Faktisk)

30 april 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

15 juli 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

13 juli 2026

Senast verifierad

1 juli 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Andra studie-ID-nummer

  • CL2-210-02
  • 2025-522343-18-00 (Ctis)

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Nej

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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