Um estudo de fase 2 de ONO-2808 em pacientes com atrofia de múltiplos sistemas
Um estudo de fase 2, duplo-cego, controlado por placebo, de grupos paralelos para avaliar a segurança, tolerabilidade, farmacocinética, farmacodinâmica e eficácia potencial de doses múltiplas de ONO-2808 em pacientes com atrofia sistêmica múltipla (MSA)
This is a Phase 2, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of multiple doses of ONO-2808 in patients with MSA. This is the first study of ONO-2808 in patients with MSA.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Descrição detalhada
This study will consist of a core part, an extension part, and an additional extension part. The purpose of the core part is to evaluate 3 doses of ONO-2808 compared to placebo in MSA patients, including: 1) safety and tolerability, 2) pharmacokinetics, and 3) changes in clinical outcome assessments (COA) and biomarkers considered to be related to the pharmacodynamics and potential efficacy of ONO-2808. The purpose of the extension part and the additional extension part is to evaluate 3 doses of ONO-2808 in MSA patients, including: 1) safety and tolerability and 2) changes in COAs and biomarkers considered to be related to the pharmacodynamics and potential efficacy of ONO-2808.
Tipo de estudo
Inscrição (Real)
Estágio
- Fase 2
Contactos e Locais
Locais de estudo
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California
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Fountain Valley, California, Estados Unidos, 92708
- The Parkinson's Movement and Disorder Institute
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Los Angeles, California, Estados Unidos, 90033
- University of Southern California
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Los Angeles, California, Estados Unidos, 90095
- David Geffen School of Medicine at UCLA
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Palo Alto, California, Estados Unidos, 94304
- Stanford University School of Medicine
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Colorado
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Englewood, Colorado, Estados Unidos, 80113
- CenExel Rocky Mountain Clinical Research
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Connecticut
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New Haven, Connecticut, Estados Unidos, 06519
- Yale School of Medicine - Yale Church Street Research Unit (CRSU)
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Florida
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Boca Raton, Florida, Estados Unidos, 33486
- Parkinson's Disease and Movement Disorders Center of Boca Raton
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Gainesville, Florida, Estados Unidos, 32608
- Norman Fixel Institute for Neurological Diseases - University of Florida
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Jacksonville, Florida, Estados Unidos, 32224
- Mayo Clinic in Florida
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Miami, Florida, Estados Unidos, 33136
- University of Miami Miller School of Medicine
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Georgia
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Atlanta, Georgia, Estados Unidos, 30329
- Emory University School of Medicine
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Kansas
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Kansas City, Kansas, Estados Unidos, 66160
- University of Kansas Medical Center Research Institute
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Massachusetts
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Boston, Massachusetts, Estados Unidos, 02115
- Brigham and Women's Hospital
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Boston, Massachusetts, Estados Unidos, 02114
- Massachusetts General Hospital
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48109
- University of Michigan School of Medicine
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Farmington Hills, Michigan, Estados Unidos, 48334
- Quest Research Institute
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Minnesota
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Rochester, Minnesota, Estados Unidos, 55905
- Mayo Clinic - Rochester
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Nebraska
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Omaha, Nebraska, Estados Unidos, 68198
- University of Nebraska Medical Center
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New York
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New York, New York, Estados Unidos, 10032
- Columbia University
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New York, New York, Estados Unidos, 10019
- Icahn School of Medicine at Mount Sinai
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New York, New York, Estados Unidos, 10016
- NYU Langone Health - NYU Dysautonomia Center
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45219
- University of Cincinnati College of Medicine
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Cleveland, Ohio, Estados Unidos, 44195
- Cleveland Clinic
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Columbus, Ohio, Estados Unidos, 43201
- Ohio State University
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Pennsylvania
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Hershey, Pennsylvania, Estados Unidos, 17033
- Penn State University - Milton S. Hershey Medical Center
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Philadelphia, Pennsylvania, Estados Unidos, 19107
- The University of Pennsylvania - Pennsylvania Hospital
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Texas
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Dallas, Texas, Estados Unidos, 75390
- UT Southwestern Medical Center
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Virginia
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Richmond, Virginia, Estados Unidos, 23298
- Virginia Commonwealth University Medical Center
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Washington
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Kirkland, Washington, Estados Unidos, 98034
- Evergreen Health
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Seattle, Washington, Estados Unidos, 98122
- Swedish Neuroscience Institute, Movement Disorders Clinic
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Aichi-ken
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Toyoake, Aichi-ken, Japão
- Fujita Health University Hospital
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Kyoto
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Kyoto, Kyoto, Japão
- National Hospital Organization Utano National Hospital
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Miyagi
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Sendai, Miyagi, Japão
- National Hospital Organization Sendai Nishitaga Hospital
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Osaka
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Toyonaka, Osaka, Japão
- National Hospital Organization Osaka Toneyama Medical Center
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Tokyo
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Fuchū, Tokyo, Japão
- Tokyo Metropolitan Neurological Hospital
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
Inclusion Criteria:
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Female or male patients with a diagnosis of clinically-established or clinically-probable MSA according to the novel Movement Disorder Society (MDS) criteria for MSA diagnosis (2022), including patients with MSA of either subtype (MSA-P or MSA-C).
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Patients at the early stages of the disease, defined as a maximum of 5 years since the onset of one of the following symptoms associated with MSA:
- Parkinsonism
- Ataxia
- Orthostatic hypotension and/or urinary dysfunction
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Patients with an anticipated survival of at least 3 years in the opinion of the Investigator.
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Patients who are able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps and then to turn around and walk at least another 10 steps. Use of assistive devices (e.g., walker or cane) is allowed.
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Ability to swallow oral medication and be willing to adhere to the study intervention regimen.
Exclusion Criteria:
- Pregnant or lactating females.
- Patients with a clinically-significant or unstable medical or surgical condition other than MSA that, in the opinion of the Investigator, might preclude safe completion of the study or might affect the results of the study (e.g., pulmonary, cardiovascular [including bradyarrhythmia], macular edema, and significant renal or hepatic dysfunction).
- Neurological diseases/disorders other than MSA, such as Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, normal pressure hydrocephalus, pharmacological, or post-encephalitic parkinsonism.
- Patients with documented liver diseases or cirrhosis.
- Positive results at Screening for active viral infections that include positive human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg) and hepatitis B core antibody, and hepatitis C virus (HCV).
- Patients with suicide ideation according to the Investigator's clinical judgment per the Columbia Suicide Severity Rating Scale (C-SSRS) at Screening or who have made a suicide attempt in the 6 months before Screening.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Triplo
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
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Comparador de Placebo: Braço placebo
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Oral administration of placebo once daily for 24 weeks in the core part and for a total of 32 weeks including the extension part; Transition to low-dose of ONO-2808 for remainder of the extension part until week 80 and for up to an additional 72 weeks in the additional extension part |
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Experimental: Braço ONO-2808
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Oral administration of ONO-2808 at low, middle or high doses once daily for 24 weeks in the core part, for a total of 80 weeks including the extension part, and for up to an additional 72 weeks in the additional extension part |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Incidence of Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Prazo: From screening up to follow-up for the core part (up to Week 28)
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A TEAE was defined as any adverse event occurring or worsening on or after the first dose of study treatment. A TEAE was considered study treatment-related if there was a reasonable causal relationship between study intervention administration and the TEAE. A serious adverse event (SAE) was an event that resulted in death, was life-threatening, required hospitalization or prolongation of hospitalization, resulted in significant disability/incapacity, was a congenital anomaly/birth defect, or was considered an important medical event. |
From screening up to follow-up for the core part (up to Week 28)
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Incidence of TEAEs by Highest Severity
Prazo: From screening up to follow-up for the core part (up to Week 28)
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A TEAE was defined as any adverse event occurring or worsening on or after the first dose of study treatment. |
From screening up to follow-up for the core part (up to Week 28)
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Incidence of Study Treatment-related TEAEs by Highest Severity
Prazo: From screening up to follow-up for the core part (up to Week 28)
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A TEAE was defined as any adverse event occurring or worsening on or after the first dose of study treatment. A TEAE was considered study treatment-related if there was a reasonable causal relationship between study intervention administration and the TEAE. |
From screening up to follow-up for the core part (up to Week 28)
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Plasma Concentration of ONO-2808
Prazo: Week 2, Week 8, Week 12, and Week 24
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Descriptive summary statistics were calculated for ONO-2808 plasma concentrations, by dose level and time point. |
Week 2, Week 8, Week 12, and Week 24
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Diretor de estudo: Project Leader, Ono Pharmaceutical Co., Ltd.
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Real)
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
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- ONO-2808-03
- jRCT2041230153 (Identificador de registro: Japan Registry of Clinical Trials)
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