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Идёт набор NCT07142343

A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease

Фаза IV С лечением CHAPLE Disease

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают
В протоколе указаны: Pozelimab.
Кому может быть актуально
Состояния в реестре: CHAPLE Disease. Базовые параметры: 1 год — 5 лет · Все.
Что важно проверить
Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится
Turkey (Türkiye)
Следующий шаг
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Официальное название

An Open-Label, Single-Arm Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Pozelimab in Pediatric Patients 1 to 5 Years of Age With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)

Обзор

This study is researching a drug called pozelimab (called "study drug"). The main aim of this study is to monitor the safety and tolerability of the study drug. The study is focused on young children 1 to 5 years of age, who have CHAPLE disease. CHAPLE is a very rare hereditary disease that can cause potentially life-threatening symptoms related to the stomach and intestines (gastrointestinal symptoms), and symptoms related to the heart and blood vessels (cardiovascular symptoms). The study is also looking at several other research questions, including: * What side effects may happen from taking the study drug * How much study drug is in the blood at different times * Whether the study drug blocks Complement 5 (C5) in the body * Whether the study drug changes the level of a substance called CH50 measured in the blood * Whether the study drug changes the levels of albumin and other proteins * Whether the body makes antibodies against study drug, which could make the study drug less effective or could lead to side effects

Вмешательства

  • Препарат Pozelimab
    Administered per the protocol

Первичные конечные точки

  • Incidence of Treatment-Emergent Adverse Event (TEAEs) [Срок оценки: Through week 52]
  • Severity of TEAEs [Срок оценки: Through week 52]
Вторичные конечные точки (12)
  • Concentrations of total pozelimab [Срок оценки: Through week 52]
  • Concentrations of total C5 in serum [Срок оценки: Through week 52]
  • Absolute change from baseline of albumin concentration in serum [Срок оценки: Through week 52]
  • Percent change from baseline of albumin concentration in serum [Срок оценки: Through week 52]
  • Absolute change from baseline of total protein [Срок оценки: Through week 52]
  • Percent change from baseline of total protein [Срок оценки: Through week 52]
  • Absolute change from baseline of total Immunoglobulin G (IgG) [Срок оценки: Through week 52]
  • Percent change from baseline of total IgG [Срок оценки: Through week 52]
  • Incidence of Anti-Drug Antibody (ADA) to pozelimab over time [Срок оценки: Through week 52]
  • Titer of ADA to pozelimab over time [Срок оценки: Through week 52]
  • Change from baseline of total complement activity CH50 over time [Срок оценки: Through week 52]
  • Percent change from baseline of CH50 over time [Срок оценки: Through week 52]

Критерии участия

Критерии включения

  • Clinical diagnosis of CD55-deficient CHAPLE disease as described in the protocol
  • Parent(s)/legal guardian(s) are willing and able to comply with participant's clinic visits and study-related procedures, including participant's completion of the full series of meningococcal vaccinations required per protocol.
  • Parent(s)/legal guardian(s) are willing to provide written informed consent
  • Participant has active CHAPLE disease as described in the protocol

Критерии исключения

  • History of meningococcal infection
  • No documented meningococcal quadrivalent (serotype ACWY) vaccination prior to screening and participant's parent(s)/legal guardian(s) are unwilling for participant to undergo vaccination during the study as described in the protocol
  • No documented vaccination for Haemophilus influenzae and Streptococcus pneumoniae if applicable based on local practice or guidelines prior to screening as described in the protocol
  • Prior treatment with a complement inhibitor as described in the protocol
  • Presence of a concomitant disease that leads to hypoproteinemia or secondary intestinal lymphangiectasia as described in the protocol

Note: Other Protocol Defined Inclusion/ Exclusion Criteria 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

Turkey (Türkiye) · 1 центр
  • Marmara University Hospital — Istanbul

Идентификаторы

NCT: NCT07142343 · R3918-PLE-2399

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗