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 de julio de 2026 actualizado por: 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.
Descripción general del estudio
Estado
Reclutamiento
Condiciones
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Intervencionista
Inscripción (Estimado)
28
Fase
- Fase 2
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Estudio Contacto
- Nombre: Patrick Round
- Número de teléfono: 888-209-5458
- Correo electrónico: clinicaltrials@rycarma.com
Ubicaciones de estudio
-
-
Baden-Wurttemberg
-
Ulm, Baden-Wurttemberg, Alemania, 89081
- Aún no reclutando
- Universitätsklinikum Ulm
-
-
State of Berlin
-
Berlin, State of Berlin, Alemania, 13125
- Reclutamiento
- Charité - Campus Berlin Buch
-
-
-
-
Barcelona
-
Barcelona, Barcelona, España, 8035
- Aún no reclutando
- Hospital Universitario Vall d'Hebron - PPDS
-
-
Guipúzcoa
-
San Sebastián, Guipúzcoa, España, 20014
- Aún no reclutando
- Hospital Universitario de Donostia
-
-
-
-
-
Paris, Francia, 73013
- Aún no reclutando
- Institut de Myologie - Hôpital de La Pitié-Salpétrière
-
-
Bouches-du-Rhône
-
Marseille, Bouches-du-Rhône, Francia, 13385
- Aún no reclutando
- AP-HM- Hôpital de La Timone
-
-
-
-
Gelderland
-
Nijmegen, Gelderland, Países Bajos, 6500 HB
- Aún no reclutando
- Radboud Universitair Medisch Centrum
-
-
-
-
-
London, Reino Unido, WC1N 3BG
- Reclutamiento
- University College Hospital - PPDS
-
-
Shropshire
-
Oswestry, Shropshire, Reino Unido, SY10 7AG
- Aún no reclutando
- The Robert Jones and Agnes Hunt Orthopaedic Hospital
-
-
Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
No
Descripción
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
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación cruzada
- Enmascaramiento: Doble
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
|
Experimental: Grupo A
|
300 mg administered once a day
Otros nombres:
|
|
Comparador de placebos: Grupo B
|
administered once a day
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Periodo de tiempo |
|---|---|
|
Change from baseline in the 1-minute sit-to-stand test (1-MSST)
Periodo de tiempo: Day 1 to day 28 [approximately]
|
Day 1 to day 28 [approximately]
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Change from baseline in the 6-Minute Walk Test (6-MNWT)
Periodo de tiempo: Day 1 to day 28 [approximately]
|
Day 1 to day 28 [approximately]
|
|
|
Change from baseline in the Timed Up and Go Test (TUG)
Periodo de tiempo: Day 1 to day 28 [approximately]
|
Day 1 to day 28 [approximately]
|
|
|
Change from baseline in the 4-Stair Climb Test (4-SCT)
Periodo de tiempo: Day 1 to day 28 [approximately]
|
Day 1 to day 28 [approximately]
|
|
|
Change from baseline Quantitative Muscle Assessment (QMA)
Periodo de tiempo: Day 1 to day 28 [approximately]
|
Day 1 to day 28 [approximately]
|
|
|
Change from baseline Manual Muscle Testing (MMT)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: Day 1 to day 28 [approximately]
|
Day 1 to day 28 [approximately]
|
|
|
Chage in International Physical Activity Questionnaire (IPAQ)
Periodo de tiempo: Day 1 to day 28 [approximately]
|
Day 1 to day 28 [approximately]
|
|
|
Number of Adverse Events
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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]
|
Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Estimado)
1 de julio de 2026
Finalización primaria (Estimado)
27 de agosto de 2027
Finalización del estudio (Estimado)
27 de agosto de 2028
Fechas de registro del estudio
Enviado por primera vez
7 de abril de 2026
Primero enviado que cumplió con los criterios de control de calidad
23 de abril de 2026
Publicado por primera vez (Actual)
30 de abril de 2026
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
15 de julio de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
13 de julio de 2026
Última verificación
1 de julio de 2026
Más información
Términos relacionados con este estudio
Palabras clave
Otros números de identificación del estudio
- CL2-210-02
- 2025-522343-18-00 (Ctis)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
NO
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
No
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .