Safety, Tolerability, Pharmacokinetics, and Efficacy of Filgotinib for the Treatment of Polyarticular-course Juvenile Idiopathic Arthritis in Children and Adolescents

Официальное название

Multicenter, Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Filgotinib in Children and Adolescents From 8 Years to Less Than 18 Years of Age With Polyarticular-course Juvenile Idiopathic Arthritis

Обзор

This is a multicenter Phase 3, open-label, single-arm study to evaluate the safety, tolerability, PK, and efficacy of orally administered filgotinib for up to 18 weeks.

Вмешательства

• Препарат Filgotinib
  IP will be provided as commercially developed film-coated tablets or age-appropriate film- coated tablets for use in paediatric subjects aged at least 8 years and needs to be taken orally q.d. at approximately the same time every morning (with or without food)

Первичные конечные точки

• Frequency and severity of treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (SAEs), and TEAEs leading to treatment discontinuation [Срок оценки: From baseline (Day 1) the study up to Week 22]

Вторичные конечные точки (9)

• Percentage of subjects with juvenile idiopathic arthritis (JIA) American College of Rheumatology (ACR) 30 response [Срок оценки: Week 12 and Week 18]
• Percentage of subjects with JIA ACR inactive disease [Срок оценки: Week 12 and Week 18]
• Change from baseline in Juvenile Arthritis Disease Activity Score (JADAS)-27 erythrocyte sedimentation rate (ESR) [Срок оценки: Week 12 and Week 18]
• Change from baseline in Juvenile Arthritis Disease Activity Score JADAS-27 C-reactive protein (CRP) [Срок оценки: Week 12 and Week 18]
• Incidence of uveitis at various timepoints (including occurrence, type, and severity) [Срок оценки: Week 1, Week 4, Week 8, Week 12, Week 18]
• PK parameters of filgotinib and its primary metabolite GS-829845 including maximum observed plasma concentration at steady-state [Cmax,ss] [Срок оценки: Week 4, Week 12 and Week 18]
• PK parameters of filgotinib and its primary metabolite GS-829845 including area under the plasma concentration-time curve over the dosing interval at steady-state [AUC0-24,ss] [Срок оценки: Week 4, Week 12 and Week 18]
• PK parameters of filgotinib and its primary metabolite GS-829845 including area under the plasma concentration-time curve over the dosing interval at steady-state for the effective exposure [AUCeff,ss] [Срок оценки: Week 4, Week 12 and Week 18]
• Acceptability of the age-appropriate pediatric formulation and the adult commercially developed film-coated tablet formulation assessed by Pediatric Oral Medicine Acceptability Questionnaire for Patients (POMAQ-P) [Срок оценки: Week 4 and Week 18]

Критерии участия

Критерии включения

• Subject and/or parent/legal guardian must be able and willing to comply with the clinical study protocol requirements and must sign and date the ICF and assent (if required per local regulation) as approved by the Independent Ethics Committee / Institutional Review Board, prior to any screening evaluations.
• Female or male subject 8 to <18 years of age, on the date of signing the informed consent and assent (per local regulation) form.
• Subject must meet the ILAR classification and have moderately to severely active disease for one of the following categories that is not adequately controlled with his/her current therapy (see Protocol Appendix 1 for disease activity assessment criteria):

• Extended oligoarthritis (i.e. affecting a total of more than 4 joints after the first 6 months of disease)
• RF-positive polyarthritis
• RF-negative polyarthritis
• PsA
• ERA
• Subject with intolerance or a history of inadequate response to at least one of the following medications for the treatment of pJIA, administered for at least 3 months, based on current treatment guidelines: conventional synthetic disease modifying anti rheumatic drugs (csDMARDs; including methotrexate) and/or biologic disease modifying anti-rheumatic drugs (bDMARDs) administered per local label, and/or non steroidal anti-inflammatory drugs for ERA and PsA subtypes.
• Female subject of childbearing potential who is sexually active and at risk for pregnancy must agree to use contraception/preventive exposure measures as described in the protocol.

Критерии исключения

• Subject with a body weight <15 kg.
• Subject with persistent oligoarthritis (i.e. affecting not more than 4 joints throughout the disease course).
• Subject with undifferentiated arthritis.
• Subject with anterior uveitis (active or uncontrolled) ≤12 weeks prior to baseline.
• Subject with systemic JIA.
• Subject with any other rheumatic disease, inflammatory, or immunologic disease (e.g. inflammatory bowel disease, hypogammaglobulinemia, or systemic lupus erythematosus).
• Subject has any condition or circumstances (including abnormalities in laboratory parameters) that, in the opinion of the investigator, may make a subject unlikely or unable to complete the study or comply with study procedures and requirements.
• Subject has an active infection. • Subject with a history of complicated herpes zoster infection (with multi dermatomal, disseminated, ophthalmic, or central nervous system involvement).

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

Польша · 3 центра

• Centrum Medyczne Hipokrates S C Elzbieta I Grzegorz Grzesk — Bydgoszcz
• MICS Centrum Medyczne Torun — Torun
• ETG JustMed — Warsaw

Великобритания · 3 центра

• Bristol Royal Hospital for Children — Bristol
• Leicester Royal Infirmary — Leicester
• Nottingham University Hospitals Queen's Medical Centre — Nottingham

Германия · 2 центра

• Charité - Campus Virchow-Klinikum — Berlin
• Hamburger Zentrum fuer Kinder und Jugendrheumatologie — Hamburg

Бельгия · 1 центр

• UZ Leuven — Leuven

Чехия · 1 центр

• Vseobecna fakultni nemocnice v Praze — Prague

Франция · 1 центр

• Hôpital Bicêtre — Le Kremlin-Bicêtre

Венгрия · 1 центр

• Pecsi Tudomanyegyetem Klinikai Kozpont — Pécs

Италия · 1 центр

• Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS — Genova

Испания · 1 центр

• Hospital Sant Joan de Deu — Barcelona

Идентификаторы

NCT: NCT07554495 · GLPG0634-CL-329 · 2024-511593-70-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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