A Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)

Официальное название

A Phase III, Multicenter, Open-Label, Uncontrolled Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)

Обзор

This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.

Вмешательства

• Препарат Satralizumab
  Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period.

Первичные конечные точки

• Summary of observed serum concentration [Cthrough] of satralizumab [Срок оценки: Week 48]
• Apparent clearance [CL/F] of satralizumab [Срок оценки: Week 48]
• Apparent volume of distribution [V/F] of satralizumab [Срок оценки: Week 48]
• Area under the concentration-time curve [AUC] of satralizumab [Срок оценки: Week 48]

Вторичные конечные точки (9)

• Proportion of relapse-free patients by Week 48 [Срок оценки: Week 48]
• Annualized relapse rate (ARR), defined as the average number of relapses for each year of the study [Срок оценки: Week 48]
• Time to first relapse (TFR) after randomization, defined as the time from randomization until the first occurrence of relapse, as determined by the investigator [Срок оценки: Week 48]
• Time to relapse requiring rescue therapy [Срок оценки: Week 48]
• Change from baseline in Expanded Disability Status Scale (EDSS) at Weeks 24 and 48 [Срок оценки: Baseline, Week 24, Week 48]
• Change from baseline in visual acuity at Weeks 24 and 48 [Срок оценки: Baseline, Week 24, Week 48]
• Change from baseline in FACES Pain Rating Scale at Weeks 24 and 48 [Срок оценки: Baseline, Week 24, Week 48]
• Change from baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) score and its proxy at Weeks 24 and 48 [Срок оценки: Baseline, Week 24, Week 48]
• Incidence and severity of adverse events [Срок оценки: Week 48]

Критерии участия

Критерии включения

• Age at screening 2-11 years, inclusive
• Body weight at screening >=10 kg
• For female patients of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception
• Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria Clinical evidence of at least one documented attack (including first attack) in the last year prior to screening
• Neurological stability for >=30 days prior to both screening and baseline
• Expanded Disability Status Scale (EDSS) 0 to 6.5
• For patients receiving a baseline immunosuppressant treatment and planning to continue on these therapies, treatment must be at stable dose for 4 weeks prior to baseline

Критерии исключения

• Pregnancy or lactation
• Evidence of other demyelinating disease mimicking NMOSD
• Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection at baseline
• Evidence of chronic active hepatitis B or C
• Evidence of untreated latent or active tuberculosis (TB)
• Receipt of a live or live-attenuated vaccine within 6 weeks prior to baseline
• History of severe allergic reaction to a biologic agent

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Аргентина · 2 центра

• Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan — Ciudad Autonoma Buenos Aires
• Clinica Universitaria Reina Fabiola — Córdoba

Китай · 2 центра

• Guangzhou Women and Children's Medical Center — Гуанчжоу
• Children's Hospital of Fudan University — Шанхай

Италия · 2 центра

• IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN — Rome
• Fondazione Istituto Neurologico Mondino IRCCS — Pavia

Польша · 2 центра

• Uniwersyteckie Centrum Kliniczne — Gdansk
• Instytut "Pomnik - Centrum Zdrowia Dziecka" — Warsaw

США · 1 центр

• Children's Hospital Colorado. — Denver

Франция · 1 центр

• Centre Hospitalier Universitaire de Bicêtre — Le Kremlin-Bicêtre

Мексика · 1 центр

• Grupo Medico Camino — DF

Turkey (Türkiye) · 1 центр

• Kocaeli University Research and Application Hospit — Kocaeli

Великобритания · 1 центр

• Great Ormond Street Hospital for Children — London

Идентификаторы

NCT: NCT05199688 · WN41733 · 2019-004092-39 · 2023-507817-85-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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