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

NCT06431776 · tracked via the Priya Life Science Germany tracker

Sponsor

Savara Inc

Phase

Phase 3

Started

2025-10-22

Last updated

2026-02-19

Condition(s) studied

Autoimmune Pulmonary Alveolar Proteinosis

Investigational drug(s) / intervention(s)

Molgramostim →

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

Study 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.

Eligibility

Sex

ALL

Min age

6 Years

Max age

18 Years

Healthy volunteers

Inclusion Criteria: * 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. Exclusion Criteria: * 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

Primary outcome measure(s)

DLCO — 24 weeks
Change in Hb-adjusted % predicted DLCO from Baseline.

Trial sites (1)

Facility	City	Region	Status
Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik	München	Germany	Recruiting

More Savara Inc. trials in Germany

Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Active, not recruiting

Other trials for the same condition

Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06431776 on ClinicalTrials.gov ↗ ← All trials in Germany