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