Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
Recruiting now · Phase 3
Conditions studied: Autoimmune Pulmonary Alveolar Proteinosis
In brief
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.
Key facts
- Study ID
- NCT06431776
- Run by
- Savara Inc.
- People needed
- 5
- Starts
- 2025-10-22
- Expected to finish
- 2027-12-01
- Last updated by the study team
- 2026-02-19
Who can join
Age: 6 and older, up to 18. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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
Where it is running
- Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik — München, Germany (enrolling)
Full record on ClinicalTrials.gov
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