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).

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.

Study Overview

• Status: Recruiting
• Conditions: Autoimmune Pulmonary Alveolar Proteinosis
• Intervention / Treatment: Drug: Molgramostim

Detailed 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.

• Study Type: Interventional
• Enrollment (Estimated): 5
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Yasmine Wasfi, MD, PhD,; Phone Number: 1 512 851 1364; Email: yasmine.wasfi@savarapharma.com
• Study Contact Backup: Name: Brian Maurer; Phone Number: 1 512 774 5786; Email: brian.mauer@savarapharma.com

Study Locations

• Germany
  • München, Germany, 80337
    • Recruiting
    • Ludwig Maximilians Universität München - Dr. von Haunersches Kinderspital - Kinderklinik und Kinderpoliklinik
    • Contact: Matthias Griese, MD; Phone Number: +49 8951603677; Email: matthias.griese@med.uni-muenchen.de
    • Contact: Angelika Gold; Phone Number: +49 894400-57871; Email: Angelika.Gold@med.uni-muenchen.de

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: molgramostim; Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.	Drug: Molgramostim; Molgramostim nebulizer solution will be administered once daily using a proprietary nebulizer optimized for the delivery of high molecular weight biologic compounds.; Other Names: Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
DLCO	Change in Hb-adjusted % predicted DLCO from Baseline.	24 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
DLCO	Change in Hb-adjusted % predicted DLCO from Baseline .	48-weeks
6-minute walk distance	Absolute change from Baseline in 6-minute walk distance (6MWD)	24-weeks
6-minute walk distance	Absolute change from Baseline in 6-minute walk distance (6MWD).	48-weeks
PedsQL	Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score.	24-weeks
PedsQL	Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score.	48-weeks
Oxygen Saturation (SpO2)	Absolute change from Baseline in oxygen saturation (SpO2)	24 weeks
Oxygen Saturation (SpO2)	Absolute change from Baseline in oxygen saturation (SpO2)	48 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Adverse events (AEs), including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain).	48 weeks
Anti-GM-CSF Ab titer	Titers of anti-GM-CSF antibodies	0, 4, 12,24,48 and 52 weeks
FEV1	Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted)	24 and 48-weeks
FVC	Change from Baseline in Forced vital capacity (FVC) (% predicted)	24 and 48-weeks

Collaborators and Investigators

• Sponsor: Savara Inc.
• Investigators: Principal Investigator: Matthias Griese, MD,, Ludwig Maximilians Universität München

Study record dates

Study Major Dates

• Study Start (Actual): October 22, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: May 22, 2024
• First Submitted That Met QC Criteria: May 22, 2024
• First Posted (Actual): May 29, 2024

Study Record Updates

• Last Update Posted (Actual): February 19, 2026
• Last Update Submitted That Met QC Criteria: February 17, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: children; GM-CSF; autoimmune; alveolar proteinosis; lung lavage
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Pulmonary Alveolar Proteinosis; Pulmonary Alveolar Proteinosis, Acquired; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Carbohydrates; Intercellular Signaling Peptides and Proteins; Glycoproteins; Glycoconjugates; Hematopoietic Cell Growth Factors; Cytokines; Colony-Stimulating Factors; regramostim; molgramostim
• Other Study ID Numbers: SAV006-04

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No