A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
A Randomized, Open-label Phase 2 Trial Consisting of a 24-Week Core Phase Followed by a 72-Week Extension Phase to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat Oral Solution in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy (DMD)
This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy (DMD). Participants will be randomized to one of three tegacorat treatment groups during a 24-week Core Phase. Participants who complete the Core Phase will continue into a 72-week Extension Phase. The study will assess muscle function, ambulatory performance, safety outcomes, pharmacokinetics, pharmacodynamic biomarkers, and health-related quality of life over a total treatment period of up to 96 weeks.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
DMD is a severe, progressive neuromuscular disorder caused by mutations in the dystrophin gene, resulting in muscle degeneration and loss of function. Current standard-of-care glucocorticoid therapy may slow disease progression but is associated with significant long-term adverse effects. Tegacorat is a selective glucocorticoid receptor agonist and modulator (SEGRAM) that has demonstrated anti-inflammatory activity with reduced glucocorticoid receptor-mediated transactivation in clinical and nonclinical studies.
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 2
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Grünenthal Clinical Trial Helpdesk
- Número de teléfono: +49 241 569 3223
- Correo electrónico: clinical-trials@grunenthal.com
Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
INCLUSION CRITERIA:
- Male participant aged ≥4 years and <7 years at randomization.
- Clinically and genetically confirmed diagnosis of Duchenne muscular dystrophy.
- Body weight between 13 kg and 35 kg at screening.
- Able to walk independently without assistive devices.
- Able to complete the TTSTAND assessment without assistance in less than 10 seconds.
- Evidence of varicella immunity by serology or vaccination history.
- Parent(s)/legal guardian(s) provide informed consent and participant assent as required.
EXCLUSION CRITERIA:
- Moderate or severe renal impairment.
- Moderate or severe hepatic impairment.
- Clinically significant abnormal laboratory findings.
- Chronic or latent infection that may increase study risk.
- Acute illness that could affect safety.
- Symptomatic cardiomyopathy.
- Diabetes mellitus.
- Severe behavioral, cognitive, or psychiatric disorders interfering with study participation.
- Adrenal insufficiency.
- Clinically significant ECG abnormalities.
- Hypersensitivity to study intervention or its components.
- Current or recent use of prohibited treatments or investigational products.
- Prior exposure to delandistrogene moxeparvovec.
- Participation in another clinical trial within the protocol-defined washout period.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
|
Experimental: Tegacorat Low dose
Tegacorat Treatment Group 1 |
Oral solution |
|
Experimental: Tegacorat Mid Dose
Tegacorat Treatment Group 2 |
Oral solution |
|
Experimental: Tegacorat High dose
Tegacorat Treatment Group 3 |
Oral solution |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Periodo de tiempo: Baseline to Week 24
|
Change from baseline to Week 24 in TTSTAND velocity. |
Baseline to Week 24
|
|
Extension Phase: Incidence of Adverse Events
Periodo de tiempo: First dose through Week 96
|
Safety and Tolerability. |
First dose through Week 96
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Periodo de tiempo: Baseline to Week 24
|
Change from baseline to Week 24 in SV95C measured using wearable sensor technology. |
Baseline to Week 24
|
|
Core Phase: Change From Baseline in 10-Meter Walk/Run (10MWR) velocity
Periodo de tiempo: Baseline to Week 24
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 24
|
|
Core Phase: Change From Baseline in Time-to-Climb-4-Stairs (TTCLIMB) velocity
Periodo de tiempo: Baseline to Week 24
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 24
|
|
Core Phase: Incidence of Adverse Events
Periodo de tiempo: First dose through Week 24
|
Safety and Tolerability. |
First dose through Week 24
|
|
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
Periodo de tiempo: Up to Week 96
|
Time to permanent discontinuation of study treatment due to an adverse event. |
Up to Week 96
|
|
Extension Phase: Change From Baseline in Weight and BMI Z-Scores
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in weight and body mass index (BMI) Z-scores. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Cushingoid Features
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in Cushingoid features. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Glucose Metabolism
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in fasting glucose and fasting serum insulin. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in PARS-III Score
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in Personal Adjustment and Role Skills Scale (PARS-III) score. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Bone Mineral Density
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in bone mineral density assessed by dual-energy X-ray absorptiometry (DXA). |
Baseline to Week 96
|
|
Extension Phase: Vertebral Fracture Assessment
Periodo de tiempo: Baseline to Week 96
|
Assessment of vertebral fractures by lateral thoracolumbar spine X-ray. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Height Z-Score
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in height Z-score. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Bone Age
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in bone age assessed by left hand X-ray. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in TTSTAND Velocity
Periodo de tiempo: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in 10MWR Velocity
Periodo de tiempo: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in TTCLIMB Velocity
Periodo de tiempo: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in SV95C
Periodo de tiempo: Baseline to Week 96
|
Change from baseline in SV95C measured using wearable sensor technology. |
Baseline to Week 96
|
Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Estimado)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Enfermedades musculoesqueléticas
- Enfermedades del Sistema Nervioso
- Enfermedades Musculares
- Enfermedades Neuromusculares
- Enfermedades Genéticas Congénitas
- Enfermedades Genéticas, Ligadas al X
- Trastornos Musculares Atróficos
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Distrofias Musculares
- Distrofia Muscular, Duchenne
Otros números de identificación del estudio
- KF6019-02
- 2026-526326-40-00 (Ctis)
- U1111-1339-2419 (Otro identificador: Universal Trial Number)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Descripción del plan IPD
Information available on the Grünenthal Group Web Site (https://www.grunenthal.com/science/clinical-trial-transparency/data-sharing) and according to the European Federation of Pharmaceutical Industries and Associations (EFPIA) Data Sharing Principles
Tipo de información de apoyo para compartir IPD
- PROTOCOLO DE ESTUDIO
- CIF
- RSC
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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