Um estudo de fase II para avaliar a eficácia clínica, segurança e tolerabilidade do MAS825 em participantes pediátricos e adultos com doença de Still's
Um estudo de fase II de marcha aberta para avaliar a eficácia clínica, a segurança e a tolerabilidade do MAS825 em participantes pediátricos e adultos com doença de Still's
The study is a phase II trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 (arumakimig) in pediatric and adult participants with Still's disease
Visão geral do estudo
Descrição detalhada
This is a multicenter, open-label, single-arm study to assess clinical efficacy, safety and tolerability of MAS825 in participants (both pediatric and adult) with Still's disease who have had inadequate response to IL-1 and/or IL-6 or other available therapies. Participants with associated lung disease and/or macrophage activation syndrome may also be included.
This study has three periods (Screening, Treatment Period 1, and Treatment Period 2) and a total duration of approximately 16 months.
Tipo de estudo
Inscrição (Estimado)
Estágio
- Fase 2
Contactos e Locais
Contato de estudo
- Nome: Novartis Pharmaceuticals
- Número de telefone: 1-888-669-6682
- E-mail: novartis.email@novartis.com
Estude backup de contato
- Nome: Novartis Pharmaceuticals
- Número de telefone: +41613241111
Locais de estudo
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Heidelberg, Alemanha, 69120
- Recrutamento
- Novartis Investigative Site
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Münster, Alemanha, 48149
- Recrutamento
- Novartis Investigative Site
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North Rhine-Westphalia
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Sankt Augustin, North Rhine-Westphalia, Alemanha, 53757
- Recrutamento
- Novartis Investigative Site
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Ontario
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Toronto, Ontario, Canadá, M5G 1X8
- Recrutamento
- Novartis Investigative Site
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Quebec
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Montreal, Quebec, Canadá, H4A 3J1
- Retirado
- Novartis Investigative Site
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Montreal, Quebec, Canadá, H3T 1C5
- Retirado
- Novartis Investigative Site
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Madrid, Espanha, 28046
- Recrutamento
- Novartis Investigative Site
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Valencia, Espanha, 46026
- Recrutamento
- Novartis Investigative Site
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Barcelona
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Esplugues, Barcelona, Espanha, 08950
- Recrutamento
- Novartis Investigative Site
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45229
- Ativo, não recrutando
- Cincinnati Childrens Hospital
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Oregon
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Portland, Oregon, Estados Unidos, 97232
- Recrutamento
- Legacy Emanuel Research Hosp Portland
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Investigador principal:
- Daniel Joseph Kingsbury
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Contato:
- Brenna Bogle
- Número de telefone: +1 503 413 5447
- E-mail: bbogle@lhs.org
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Utah
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Salt Lake City, Utah, Estados Unidos, 84113
- Recrutamento
- University of Utah
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Contato:
- Tressie Rollins
- Número de telefone: +1 415 515 8212
- E-mail: tressie.rollins@hsc.utah.edu
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Investigador principal:
- Aimee Ogden Hersh
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Bordeaux, França, 33076
- Recrutamento
- Novartis Investigative Site
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Bron, França, 69677
- Recrutamento
- Novartis Investigative Site
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Le Kremlin-Bicêtre, França, 94275
- Recrutamento
- Novartis Investigative Site
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Lille, França, 59037
- Recrutamento
- Novartis Investigative Site
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Paris, França, 75015
- Recrutamento
- Novartis Investigative Site
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Paris, França, 75970
- Recrutamento
- Novartis Investigative Site
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Utrecht, Holanda, 3584 CX
- Ativo, não recrutando
- Novartis Investigative Site
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FI
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Florence, FI, Itália, 50139
- Recrutamento
- Novartis Investigative Site
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GE
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Genova, GE, Itália, 16147
- Recrutamento
- Novartis Investigative Site
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RM
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Roma, RM, Itália, 00165
- Recrutamento
- Novartis Investigative Site
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Fatih
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Istanbul, Fatih, Turquia (Türkiye), 34098
- Recrutamento
- Novartis Investigative Site
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Sihhiye Altindag
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Ankara, Sihhiye Altindag, Turquia (Türkiye), 06230
- Recrutamento
- Novartis Investigative Site
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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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Age ≥ 1 with a diagnosis of Still's Disease
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Active diseases defined as:
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CRP or ferritin levels greater than ULN, and any of:
- Fever ≥ 38°C attributed to Still's Disease activity and documented for a number of days prior to Day 1 or
- Rash attributed to Still's Disease activity or
- Musculoskeletal involvement: arthritis in a number of joints per ACP criteria for active joint or
- Serositis or
- Macrophage activation syndrome activity as defined by ferritin levels and at least one of: platelet count, a biomarker or fibrinogen levels attributed to Still's Disease activity by the investigator
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Intolerance or inadequate response to available biologic therapy
Exclusion Criteria:
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Patients out of weight range
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Ongoing or previous treatment with immunomodulatory drugs
- A limited number of Still's Disease patients that have previously received MAS825 through a managed access program are permitted on the study
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Glucocorticoid dose exceeding a set limit
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Any conditions or significant medical problems which places the patient at unacceptable risk for MAS825 therapy
- Still's disease patients with evidence of macrophage activation syndrome are permitted in the study
- Still's Disease patients with evidence of interstitial lung disease including those requiring supplemental oxygen therapy are permitted in the study
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History of ongoing, chronic or possibly recurrent infection (e.g. HIV, TB, HCV, HBV) and/or symptoms and signs of clinically significant active bacterial, fungal or viral infections
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Live vaccinations within a set time prior to MAS825 treatment. Live vaccines are prohibited up to several months following the last dose
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History of malignancy of any organ system, including post-transplant lymphoproliferative disorder, treated or untreated, within a number of years, regardless of whether there is evidence of local recurrence and metastases
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History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients
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Pregnant or breastfeeding women
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Women of child-bearing potential who do not agree to comply with required contraceptive use
Other protocol-defined inclusion/exclusion criteria may apply.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
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Experimental: MAS825
Experimental drug |
Experimental drug
Outros nomes:
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
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Number of participants with clinical response based on one set of response criteria
Prazo: Day 85
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Established measure for efficacy of treatments in the rheumatologic conditions taking into account self-assessment, function and survival. |
Day 85
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Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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Number of participants with clinical response based on another set of response criteria
Prazo: Day 85
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Established measure for efficacy of treatments in the rheumatologic conditions taking into account self-assessment, function and survival. |
Day 85
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Response based on a biomarker level
Prazo: Baseline, Day 85
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To evaluate the efficacy of MAS825 in reducing clinical disease activity |
Baseline, Day 85
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Change in physician assessment based on laboratory features of MAS825
Prazo: Baseline, 15 months
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To assess the effect of MAS825 on macrophage activation syndrome |
Baseline, 15 months
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Change from baseline in Glucocorticoid (GC) dose
Prazo: Baseline, Day 85, Month 6, Month 12
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To evaluate the efficacy of MAS825 as a glucocorticoid-sparing agent |
Baseline, Day 85, Month 6, Month 12
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Change from baseline in patient/parent assessment of physical function as assessed by Quality of Life instrument
Prazo: Baseline, Day 85
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To evaluate the efficacy of MAS825 in improving participant's quality of life |
Baseline, Day 85
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Clinically inactive disease whilst on non-treatment dose corticosteroid at any time during the study
Prazo: 15 months
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The number of participants with inactive disease will be assessed. Disease activity will be evaluated by the investigator based on physical examination and laboratory parameters. Inactive disease is defined according to protocol-specific criteria, which include clinical signs, laboratory values, and physician assessment. |
15 months
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Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
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
Outros números de identificação do estudo
- CMAS825J12201
- 2024-520208-25-00 (Outro identificador: EU CT Number)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano IPD
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Informações sobre medicamentos e dispositivos, documentos de estudo
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