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

Last updated: February 17, 2026
Sponsor: Savara Inc.
Overall Status: Active - Recruiting

Phase

3

Condition

Connective Tissue Diseases

Collagen Vascular Diseases

Lung Disease

Treatment

Molgramostim

Clinical Study ID

NCT06431776
SAV006-04
  • Ages 6-18
  • All Genders

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 Criteria

Inclusion

Inclusion Criteria:

  • Be ≥6 and <18 years of age, at the time of signing the informed consent and informedassent (if applicable).

  • Have a history of pulmonary alveolar proteinosis, based on examination of a lungbiopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram ofthe 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

Exclusion Criteria:

  • Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolicdisorder of surfactant production.

  • Have undergone treatment with Lung Lavage (WLL) within 1 month of Baseline

Study Design

Total Participants: 5
Treatment Group(s): 1
Primary Treatment: Molgramostim
Phase: 3
Study Start date:
October 22, 2025
Estimated Completion Date:
December 31, 2027

Study Description

This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP).

The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology.

The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP.

Connect with a study center

  • Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik

    München 2867711, 80337
    Germany

    Active - Recruiting

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