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.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Descrição detalhada
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 estudo
Inscrição (Estimado)
Estágio
- Fase 2
Contactos e Locais
Contato de estudo
- Nome: Grünenthal Clinical Trial Helpdesk
- Número de telefone: +49 241 569 3223
- E-mail: clinical-trials@grunenthal.com
Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
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.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
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 |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Core Phase: Change From Baseline in Time-to-Stand-from-Supine (TTSTAND) Velocity
Prazo: Baseline to Week 24
|
Change from baseline to Week 24 in TTSTAND velocity. |
Baseline to Week 24
|
|
Extension Phase: Incidence of Adverse Events
Prazo: First dose through Week 96
|
Safety and Tolerability. |
First dose through Week 96
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Core Phase: Change From Baseline in Stride Velocity 95th Centile (SV95C)
Prazo: 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
Prazo: 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
Prazo: Baseline to Week 24
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 24
|
|
Core Phase: Incidence of Adverse Events
Prazo: First dose through Week 24
|
Safety and Tolerability. |
First dose through Week 24
|
|
Extension Phase: Time to Treatment Discontinuation Due to Adverse Events
Prazo: 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
Prazo: 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
Prazo: Baseline to Week 96
|
Change from baseline in Cushingoid features. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Glucose Metabolism
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: Baseline to Week 96
|
Change from baseline in height Z-score. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in Bone Age
Prazo: 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
Prazo: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in 10MWR Velocity
Prazo: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in TTCLIMB Velocity
Prazo: Baseline to Week 96
|
Change From Baseline in Functional Test Performance. |
Baseline to Week 96
|
|
Extension Phase: Change From Baseline in SV95C
Prazo: Baseline to Week 96
|
Change from baseline in SV95C measured using wearable sensor technology. |
Baseline to Week 96
|
Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Doenças musculoesqueléticas
- Doenças do Sistema Nervoso
- Doenças Musculares
- Doenças Neuromusculares
- Doenças Genéticas, Congênitas
- Doenças Genéticas, Ligadas ao X
- Distúrbios Musculares Atróficos
- Doenças e Anormalidades Congênitas, Hereditárias e Neonatais
- Distrofias Musculares
- Distrofia Muscular, Duchenne
Outros números de identificação do estudo
- KF6019-02
- 2026-526326-40-00 (Ctis)
- U1111-1339-2419 (Outro identificador: Universal Trial Number)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano 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 informação de suporte de compartilhamento de IPD
- PROTOCOLO DE ESTUDO
- CIF
- CSR
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