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RecruitingPhase 3NCT06431776

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

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

Sponsor

Savara Inc.

Enrollment

5 participants

Start Date

Oct 22, 2025

Study Type

INTERVENTIONAL

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

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

Min Age: 6 YearsMax Age: 18 Years

Inclusion Criteria4

  • 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 Criteria2

  • 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
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Interventions

DRUGMolgramostim

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

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NCT06431776