Actively Recruiting
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
Led by Savara Inc. · Updated on 2026-02-19
5
Participants Needed
1
Research Sites
110 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Be 6 to under 18 years of age at the time of consent.
- Have a history of pulmonary alveolar proteinosis confirmed by lung biopsy, bronchoalveolar lavage cytology, or high-resolution chest CT.
- Have a positive serum anti-GM-CSF autoantibody test confirming autoimmune pulmonary alveolar proteinosis.
- Have a hemoglobin-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) 70% predicted or lower at screening.
You will not qualify if you...
- Have a diagnosis of hereditary (congenital) or secondary pulmonary alveolar proteinosis, or a metabolic disorder of surfactant production.
- Have undergone whole lung lavage treatment within 1 month prior to baseline.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik
München, Germany, 80337
Actively Recruiting
Research Team
Y
Yasmine Wasfi, MD, PhD,
CONTACT
B
Brian Maurer
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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