A Phase II Trial to Evaluate the Clinical Efficacy, Safety and Tolerability of MAS825 in Pediatric and Adult Participants With Still's Disease

Официальное название

An Open-label Phase II Trial to Evaluate the Clinical Efficacy, Safety and Tolerability of MAS825 in Pediatric and Adult Participants With Still's Disease

Обзор

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

Подробное описание

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.

Вмешательства

• Препарат MAS825
  Experimental drug

Первичные конечные точки

• Number of participants with clinical response based on one set of response criteria [Срок оценки: Day 85]

Вторичные конечные точки (6)

• Number of participants with clinical response based on another set of response criteria [Срок оценки: Day 85]
• Response based on a biomarker level [Срок оценки: Baseline, Day 85]
• Change in physician assessment based on laboratory features of MAS825 [Срок оценки: Baseline, 15 months]
• Change from baseline in Glucocorticoid (GC) dose [Срок оценки: Baseline, Day 85, Month 6, Month 12]
• Change from baseline in patient/parent assessment of physical function as assessed by Quality of Life instrument [Срок оценки: Baseline, Day 85]
• Clinically inactive disease whilst on non-treatment dose corticosteroid at any time during the study [Срок оценки: 15 months]

Критерии участия

Критерии включения

• Age ≥ 1 with a diagnosis of Still's Disease
• Active diseases defined as:
• 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
• Intolerance or inadequate response to available biologic therapy

Критерии исключения

• Patients out of weight range
• 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
• Glucocorticoid dose exceeding a set limit
• 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
• 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
• Live vaccinations within a set time prior to MAS825 treatment. Live vaccines are prohibited up to several months following the last dose
• 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
• History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients
• Pregnant or breastfeeding women
• Women of child-bearing potential who do not agree to comply with required contraceptive use

Other protocol-defined inclusion/exclusion criteria may apply.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Франция · 6 центров

• Novartis Investigative Site — Bordeaux
• Novartis Investigative Site — Bron
• Novartis Investigative Site — Le Kremlin-Bicêtre
• Novartis Investigative Site — Lille
• Novartis Investigative Site — Paris
• Novartis Investigative Site — Paris

Германия · 3 центра

• Novartis Investigative Site — Sankt Augustin
• Novartis Investigative Site — Heidelberg
• Novartis Investigative Site — Münster

Италия · 3 центра

• Novartis Investigative Site — Florence
• Novartis Investigative Site — Genova
• Novartis Investigative Site — Roma

Испания · 3 центра

• Novartis Investigative Site — Esplugues
• Novartis Investigative Site — Madrid
• Novartis Investigative Site — Valencia

США · 2 центра

• Cincinnati Childrens Hospital — Cincinnati
• Legacy Emanuel Research Hosp Portland — Portland

Канада · 2 центра

• Novartis Investigative Site — Montreal
• Novartis Investigative Site — Montreal

Turkey (Türkiye) · 2 центра

• Novartis Investigative Site — Istanbul
• Novartis Investigative Site — Ankara

Нидерланды · 1 центр

• Novartis Investigative Site — Utrecht

Идентификаторы

NCT: NCT07203001 · CMAS825J12201 · 2024-520208-25

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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