Clinical Research Directory
Browse clinical research sites, groups, and studies.
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
Sponsor: Savara Inc.
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.
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).
Key Details
Gender
All
Age Range
6 Years - 18 Years
Study Type
INTERVENTIONAL
Enrollment
5
Start Date
2025-10-22
Completion Date
2027-12
Last Updated
2026-02-19
Healthy Volunteers
No
Interventions
Molgramostim
Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds.
Locations (1)
Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik
München, Germany