Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).

Официальное название

An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).

Обзор

The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are: The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP. This is an open-label study: all participants will receive treatment with molgramostim. Patients will: * Take molgramostim once daily via nebulizer every day for 12 months. * Visit the clinic approximately every 12 weeks for checkups and tests. * Keep a diary of any oxygen use.

Подробное описание

This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP).

The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology.

The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP.

Вмешательства

• Препарат Molgramostim
  Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds.

Первичные конечные точки

• DLCO [Срок оценки: 24 weeks]

Вторичные конечные точки (7)

• DLCO [Срок оценки: 48-weeks]
• 6-minute walk distance [Срок оценки: 24-weeks]
• 6-minute walk distance [Срок оценки: 48-weeks]
• PedsQL [Срок оценки: 24-weeks]
• PedsQL [Срок оценки: 48-weeks]
• Oxygen Saturation (SpO2) [Срок оценки: 24 weeks]
• Oxygen Saturation (SpO2) [Срок оценки: 48 weeks]

Критерии участия

Критерии включения

• Be ≥6 and <18 years of age, at the time of signing the informed consent and informed assent (if applicable).
• Have a history of pulmonary alveolar proteinosis, based on examination of a lung biopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram of the chest.
• Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP.
• Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening.

Критерии исключения

• Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production.
• Have undergone treatment with Lung Lavage (WLL) within 1 month of 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

Германия · 1 центр

• Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik u — München

Идентификаторы

NCT: NCT06431776 · SAV006-04

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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