A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

Официальное название

A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

Обзор

The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).

Вмешательства

• Препарат Zilucoplan
  Zilucoplan will be administered subcutaneously to pediatric study participants.

Первичные конечные точки

• Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) [Срок оценки: Week 4 (Day 29)]
• Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) [Срок оценки: Week 4 (Day 29)]
• Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29) [Срок оценки: Week 4 (Day 29)]

Вторичные конечные точки (9)

• Occurence of treatment-emergent adverse events (TEAEs) during the course of the study [Срок оценки: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
• Occurrence of treatment-emergent serious adverse events (TESAEs) [Срок оценки: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
• Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP) [Срок оценки: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
• Occurrence of treatment-emergent infections [Срок оценки: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)]
• Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29) [Срок оценки: Week 4 (Day 29)]
• Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29). [Срок оценки: Week 4 (Day 29)]
• Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29) [Срок оценки: Week 4 (Day 29)]
• Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29) [Срок оценки: Week 4 (Day 29)]
• Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29) [Срок оценки: Week 4 (Day 29)]

Критерии участия

Критерии включения

United States of America (USA) specific inclusion criterion:

\- Participant must be 12 to <18 years of age at the time of signing the Informed consent/assent according to local regulation

Rest of world (ROW) specific inclusion criterion:

\- Participant must be 2 to <18 years of age at the time of signing the Informed consent/assent according to local regulation

Global inclusion criteria:

• Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening
• Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening
• Participants with gMG, including:
• An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to <18 years of age at Screening
• Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to <12 years of age at Screening (does not apply to US)
• Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s)

Критерии исключения

• Participant has known positive serology for muscle-specific kinase
• Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study
• Participant has had a thymectomy within 6 months prior to Baseline
• Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator
• Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline

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

США · 2 центра

• Mg0014 50168 — Chicago
• Mg0014 50574 — Flower Mound

Польша · 2 центра

• Mg0014 40774 — Katowice
• Mg0014 40218 — Warsaw

South Korea · 2 центра

• Mg0014 20104 — Seoul
• Mg0014 20220 — Seoul

Великобритания · 2 центра

• Mg0014 40735 — Glasgow
• Mg0014 40736 — London

Италия · 1 центр

• Mg0014 40144 — Milan

Идентификаторы

NCT: NCT06055959 · MG0014 · U1111-1290-3349 · 2022-502072-23

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗