A Pharmacokinetics (PK), Pharmacodynamics (PD), Safety and Tolerability Study of Fenebrutinib in Children and Adolescents With Relapsing Multiple Sclerosis (RMS)

Официальное название

An Open-label, Single-arm Study to Evaluate Pharmacokinetics, Pharmacodynamic Effects, Safety and Tolerability of Fenebrutinib in Children and Adolescents With Relapsing Multiple Sclerosis

Обзор

This open label, single arm study will evaluate the PK and PD effects of fenebrutinib in children and adolescents with RMS aged between 10 and \< 18 years. This study consists of a Dose Exploration Period and an Optional Extension Period. Eligible participants may choose to continue treatment with fenebrutinib in the optional extension period after completing the dose exploration period.

Вмешательства

• Препарат Fenebrutinib
  Fenebrutinib will be administered orally.

Первичные конечные точки

• Plasma Concentration of Fenebrutinib [Срок оценки: Up to Week 96]
• Total Number of New T1 Gadolinium (Gd)-enhancing Lesions on Brain Observed Through Magnetic Resonance Imaging (MRI) Scans [Срок оценки: At Week 12]

Вторичные конечные точки (8)

• Number of Participants With Adverse Events (AEs) [Срок оценки: Up to approximately 100 weeks]
• Percentage of Participants With Suicidal Ideation (SI) or Behavior, as Assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) [Срок оценки: Up to approximately 100 weeks]
• Change From Baseline in Vital Signs - Pulse Rate [Срок оценки: Baseline up to approximately 100 Weeks]
• Change From Baseline in Vital Signs - Blood Pressure [Срок оценки: Baseline up to approximately 100 Weeks]
• Change From Baseline in Single 12-lead Electrocardiogram (ECG) Parameter - QTc Interval [Срок оценки: Baseline up to approximately 100 Weeks]
• Change From Baseline in Single 12-lead ECG Parameter - PR Interval [Срок оценки: Baseline up to approximately 100 Weeks]
• Change from Baseline in Clinical Laboratory Test Results - Alanine Aminotransferase (ALT) [Срок оценки: Baseline up to approximately 100 Weeks]
• Change from Baseline in Clinical Laboratory Test Results - Aspartate Aminotransferase (AST) [Срок оценки: Baseline up to approximately 100 Weeks]

Критерии участия

Критерии включения

• A diagnosis of RMS in accordance with the International Pediatric Multiple Sclerosis Study Group (IPMSSG) criteria for pediatric MS, Version 2012, and the revised 2017 McDonald Criteria and one or more of the following: at least one MS relapse during the previous year or two MS relapses in the previous 2 years or evidence of at least one Gd enhancing lesion on MRI within 6 month
• Expanded Disability Status Scale (EDSS) at screening from 0 to 5.5 points, inclusive
• Children and adolescents must have received all childhood vaccinations as per local/national recommendations for childhood vaccination against infectious diseases

Критерии исключения

• A diagnosis of primary progressive multiple sclerosis (PPMS) or non-active secondary progressive multiple sclerosis (SPMS)
• Co-morbid Conditions:
• Potentially confounding neurological, somatic, or metabolic disorders
• Current clinically significant psychiatric or medical illness
• History of cancer, transplants, or bleeding disorders
• Inability to complete an MRI scan or get gadolinium
• Abnormal liver function tests or blood counts
• Sensitivity or intolerance to any ingredient (including excipients) of fenebrutinib tablets
• Active, recurrent, or chronic infections
• Recent or anticipated use of prohibited medications/treatments:
• Certain disease-modifying therapy (DMT) and other immunosuppressants
• Drugs interacting with fenebrutinib (Cytochrome P450 3A4 \[CYP3A4\] inhibitors)
• Any other investigational therapy, anticoagulants, certain vaccines
• A score of 4 or 5 on the "last 6 months" section of the screening SI section or "yes" on any item of the "last 6 months" Suicidal Behavior (SB) section of the C-SSRS or a positive answer on Question 9 of the Patient Health Questionnaire-9 Modified for Adolecents (PHQ-A) or significant risk of suicide, in the investigator's judgment

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

Бразилия · 4 центра

• L2IP ?Instituto de Pesquisas Clínicas Ltda. — Brasília
• Instituto de Neurologia de Curitiba - Hospital Ecoville — Curitiba
• Nucleo de Pesquisa Clinica do Rio Grande do Sul NPCR — Porto Alegre
• Centro de Pesquisas Clinicas - CPCLIN — São Paulo

Польша · 3 центра

• Uniwersyteckie Centrum Kliniczne — Gdansk
• Uniwersytecki Szpital Kliniczny w Poznaniu — Poznan
• Instytut "Pomnik - Centrum Zdrowia Dziecka" — Warsaw

Испания · 3 центра

• Hospital Universitario de La Princesa — Madrid
• Hospital Universitario Ramon y Cajal — Madrid
• Hospital Universitario Virgen Macarena — Seville

Аргентина · 2 центра

• INECO Neurociencias Orono — Rosario
• Sanatorio del Sur S.A. — San Miguel de Tucumán

Мексика · 2 центра

• Clinstile S.A de C.V. — Mexico City
• Neurociencias Estudios Clinicos S.C. — Culiacán

Португалия · 2 центра

• Centro Clnico Acadmico - Braga, Associao (2CA-Braga) — Braga
• Unidade Local de Saude de Coimbra E P E — Coimbra

Украина · 1 центр

• UH of State NPC Danulo Halytsky NMU in Lviv CID, Clinic of Neurology and Neurosurgery — Lviv

Идентификаторы

NCT: NCT07161258 · CN45847 · 2024-519800-28-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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