Diese Seite wurde automatisch übersetzt und die Genauigkeit der Übersetzung wird nicht garantiert. Bitte wende dich an die englische Version für einen Quelltext.

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

23. April 2026 aktualisiert von: 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.

Studienübersicht

Status

Noch keine Rekrutierung

Bedingungen

Intervention / Behandlung

Detaillierte Beschreibung

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.

Studientyp

Interventionell

Einschreibung (Geschätzt)

28

Phase

  • Phase 2

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienkontakt

Studienorte

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

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Beschreibung

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

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

  • Hauptzweck: Behandlung
  • Zuteilung: Zufällig
  • Interventionsmodell: Crossover-Aufgabe
  • Maskierung: Doppelt

Waffen und Interventionen

Teilnehmergruppe / Arm
Intervention / Behandlung
Experimental: Gruppe A
Arzneimittel: Surlorian
300 mg administered once a day
Andere Namen:
  • ARM210 (S48168)
Placebo-Komparator: Gruppe B
Sonstiges: Placebo
administered once a day

Was misst die Studie?

Primäre Ergebnismessungen

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

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Change from baseline in the 6-Minute Walk Test (6-MNWT)
Zeitfenster: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in the Timed Up and Go Test (TUG)
Zeitfenster: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline in the 4-Stair Climb Test (4-SCT)
Zeitfenster: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline Quantitative Muscle Assessment (QMA)
Zeitfenster: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Change from baseline Manual Muscle Testing (MMT)
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Chage in International Physical Activity Questionnaire (IPAQ)
Zeitfenster: Day 1 to day 28 [approximately]
Day 1 to day 28 [approximately]
Number of Adverse Events
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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]

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

RyCarma Therapeutics, Inc.

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Geschätzt)

15. April 2026

Primärer Abschluss (Geschätzt)

27. August 2027

Studienabschluss (Geschätzt)

27. August 2028

Studienanmeldedaten

Zuerst eingereicht

7. April 2026

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

23. April 2026

Zuerst gepostet (Tatsächlich)

30. April 2026

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

30. April 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

23. April 2026

Zuletzt verifiziert

1. April 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Schlüsselwörter

Andere Studien-ID-Nummern

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

Plan für individuelle Teilnehmerdaten (IPD)

Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?

NEIN

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Nein

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .

Klinische Studien zur Autosomal Dominant RYR1-Related Myopathy

Suchen Sie nach ähnlichen Studien

Sponsoren und Mitarbeiter

Krankheiten

Drogeninterventionen

CROs by country

CROs in Equatorial Guinea

Bedingungen

Seltene Krankheiten

Drogeninterventionen

Nahrungsergänzungsmittel

Sponsor / Mitarbeiter

Standorte

Abonnieren