Trial Outcomes & Findings for Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP) (NCT NCT04544293)

Last Updated: 2025-08-07

Results Overview

As a measure of pulmonary gas transfer, a standardized lung function test, DLCO, was conducted. The single-breath DLCO test was performed in accordance with American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines for DLCO testing. Results reported as % predicted DLCO adjusted for hemoglobin concentration (%predicted DLCOadj).

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE3

Target enrollment

164 participants

Primary outcome timeframe

From Baseline to Week 24

Results posted on

2025-08-07

Participant Flow

Outpatient Medical Clinics 19 May 2021 -16 June 2023

Two screening visits were conducted 6 and 3 weeks prior to the Day 1 baseline visit. At the baseline visit, eligible subjects were centrally randomized through a Randomization and Trial Supply Management system to 48 weeks of double-blind treatment with MOL-OD or PBO. To be randomized patients were required to have a percent predicted DLCO adjusted for hemoglobin (%DLCOadj) of 70% or less and no variation in %DLCOadj of +/-15%.

Participant milestones

Participant milestones
Measure	Molgramostim Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Double-blind STARTED	81	83
Double-blind COMPLETED	79	80
Double-blind NOT COMPLETED	2	3
Open-Label Extension STARTED	79	81
Open-Label Extension COMPLETED	0	0
Open-Label Extension NOT COMPLETED	79	81

Reasons for withdrawal

Reasons for withdrawal
Measure	Molgramostim Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Double-blind Adverse Event	2	1
Double-blind Pregnancy	0	1
Double-blind Lost to Follow-up	0	1
Open-Label Extension Study ongoing	79	81

Baseline Characteristics

Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution	Total n=164 Participants Total of all reporting groups
Age, Continuous	50.8 years STANDARD_DEVIATION 13.03 • n=5 Participants	48.4 years STANDARD_DEVIATION 12.69 • n=7 Participants	49.6 years STANDARD_DEVIATION 12.87 • n=5 Participants
Sex: Female, Male Female	37 Participants n=5 Participants	29 Participants n=7 Participants	66 Participants n=5 Participants
Sex: Female, Male Male	44 Participants n=5 Participants	54 Participants n=7 Participants	98 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	36 Participants n=5 Participants	37 Participants n=7 Participants	73 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) White	38 Participants n=5 Participants	40 Participants n=7 Participants	78 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Region of Enrollment Romania	4 participants n=5 Participants	2 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants	12 participants n=7 Participants	23 participants n=5 Participants
Region of Enrollment Japan	29 participants n=5 Participants	24 participants n=7 Participants	53 participants n=5 Participants
Region of Enrollment United Kingdom	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Portugal	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Spain	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Canada	5 participants n=5 Participants	2 participants n=7 Participants	7 participants n=5 Participants
Region of Enrollment South Korea	6 participants n=5 Participants	10 participants n=7 Participants	16 participants n=5 Participants
Region of Enrollment Turkey	8 participants n=5 Participants	10 participants n=7 Participants	18 participants n=5 Participants
Region of Enrollment Belgium	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment Ireland	2 participants n=5 Participants	0 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Poland	0 participants n=5 Participants	3 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Italy	4 participants n=5 Participants	5 participants n=7 Participants	9 participants n=5 Participants
Region of Enrollment Australia	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment France	5 participants n=5 Participants	4 participants n=7 Participants	9 participants n=5 Participants
Region of Enrollment Germany	4 participants n=5 Participants	6 participants n=7 Participants	10 participants n=5 Participants
%DLCOadj at Baseline	54.0 % n=5 Participants	55.0 % n=7 Participants	55 % n=5 Participants
%DLCOadj at Randomization <=50%	31 Participants n=5 Participants	32 Participants n=7 Participants	63 Participants n=5 Participants
%DLCOadj at Randomization >50%	50 Participants n=5 Participants	51 Participants n=7 Participants	101 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Baseline to Week 24

Population: Full Analysis Set

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in Percent (%) Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) Adjusted for Hemoglobin Concentration to Week 24	9.8 % predicted DLCO adjusted for hemoglobin Interval 7.3 to 12.3	3.8 % predicted DLCO adjusted for hemoglobin Interval 1.4 to 6.3

SECONDARY outcome

Timeframe: From Baseline to Week 48

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in Percent (%) Predicted DLCO Adjusted for Hemoglobin Concentration (%DLCOadj) to Week 48	11.6 Unit of Measure: % predicted DLCO adjust Interval 8.7 to 14.5	4.7 Unit of Measure: % predicted DLCO adjust Interval 1.8 to 7.6

SECONDARY outcome

Timeframe: From Baseline to Week 24

The Saint Georges Respiratory Questionnaire (SGRQ) is designed to measure respiratory health impairment. The scale includes questions related to three components: Activity (activities that cause or are limited by breathlessness), Impact (social functioning and psychological disturbances resulting from airway disease), and Symptoms (respiratory symptoms, their frequency and severity). The questionnaire has a recall period of 4 weeks for Symptoms, whereas Activity and Impact components address the subject's current state. SGRQ scored on a 0-100 scale with lower scores indicating better respiratory health.

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in St. Georges Respiratory Questionnaire (SGRQ) Total Score to Week 24	-11.5 Change from baseline units on a scale Interval -15.01 to -7.98	-4.9 Change from baseline units on a scale Interval -8.28 to -1.52

SECONDARY outcome

Timeframe: From Baseline to Week 24

The Saint Georges Respiratory Questionnaire (SGRQ) Activity scale is a subscale of the SGRQ and is designed to measure the effect of respiratory health impairment on daily activity affected by breathlessness. The questionnaire for the Activity components addresses the subject's current state. SGRQ Activity is scored on a 0-100 scale with lower scores indicating better respiratory health activity

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in SGRQ Activity Component Score to Week 24	-13.03 units on a scale Interval -17.58 to -8.49	-5.22 units on a scale Interval -9.76 to -0.69

SECONDARY outcome

Timeframe: From Baseline to Week 24

As a functional measure of exertional limitations related to dyspnea, EC was assessed by an exercise treadmill test. EC was expressed in peak METs (1 MET=3.5 mL O2/kg/min). The highest treadmill speed and grade achieved was used to calculate peak METs. Higher values indicate better functional capacity.

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in Exercise Capacity (EC), Expressed as Peak Metabolic Equivalents (METs) to Week 24	1.11 Change from baseline in METS Interval 0.76 to 1.46	0.70 Change from baseline in METS Interval 0.34 to 1.05

SECONDARY outcome

Timeframe: From Baseline to Week 48

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in SGRQ Total Score to Week 48	-10.72 Change from baseline units on a scale Interval -15.01 to -6.44	-5.85 Change from baseline units on a scale Interval -9.96 to -1.74

SECONDARY outcome

Timeframe: From Baseline to Week 48

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in SGRQ Activity From Baseline to Week 48	-13.39 Change from baseline score on a scale Interval -18.87 to -7.91	-7.40 Change from baseline score on a scale Interval -12.72 to -2.09

SECONDARY outcome

Timeframe: From Baseline to Week 48

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in EC, Expressed as Peak METs to Week 48	1.13 Change from baseline in METS Interval 0.77 to 1.49	0.58 Change from baseline in METS Interval 0.22 to 0.93

SECONDARY outcome

Timeframe: From Baseline to Week 24

A-aDO2 was used as an additional measure of gas exchange.

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change From Baseline in Alveolar-arterial Oxygen Difference (A-aDO2) to Week 24 (All Subjects)	-7.97 mmHg Interval -11.62 to -4.32	-3.96 mmHg Interval -7.48 to -0.44

SECONDARY outcome

Timeframe: From Baseline until End of Double-blind treatment (Week 48)

Assessment of the safety of molgramostim compared to placebo

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Number of Subjects With Serious and Non-serious Adverse Events Any TEAE	69 participants	71 participants
Number of Subjects With Serious and Non-serious Adverse Events Any TESAE	14 participants	20 participants

SECONDARY outcome

Timeframe: From Baseline until End of Double-blind treatment Week-48

Population: One subject in the molgramostim group had missing antiGM-CSF titers.

Assess the effect of molgramostim or placebo on antiGM-CSF antibody titers that increased by a dilution factor of 2.(4X increase in titer compared to Baseline).

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Number of Subjects With Positive Treatment-boosted Anti Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) Antibody Titers During 24 Weeks' Treatment and During 48 Weeks' Treatment Week 24	28 Participants	30 Participants
Number of Subjects With Positive Treatment-boosted Anti Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) Antibody Titers During 24 Weeks' Treatment and During 48 Weeks' Treatment Week 48	39 Participants	37 Participants

SECONDARY outcome

Timeframe: From Baseline to Weeks 24 and 48

Forced vital capacity is the volume of air that can be expired after a deep breath. Higher volumes indicate better respiratory function. FVC is scored as % of predicted normal expired vital capacity.

Outcome measures

Outcome measures
Measure	Molgramostim n=78 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=81 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Changes in Forced Vital Capacity (FVC) %Predicted Normal Week 24	3.2 % FVC predicted Standard Deviation 6.20	0.9 % FVC predicted Standard Deviation 5.74
Changes in Forced Vital Capacity (FVC) %Predicted Normal Week 48	3.2 % FVC predicted Standard Deviation 7.92	-0.1 % FVC predicted Standard Deviation 8.27

SECONDARY outcome

Timeframe: From Baseline to Weeks 24 and 48

FEV1 is the volume of air expired in 1 second in liters (L). Higher values indicate better respiratory function.

Outcome measures

Outcome measures
Measure	Molgramostim n=78 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=81 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Changes in Forced Expiratory Volume in One Second (FEV1) % Predicted Normal. Week 24	1.2 %predicted normal Standard Deviation 7.08	-0.3 %predicted normal Standard Deviation 5.35
Changes in Forced Expiratory Volume in One Second (FEV1) % Predicted Normal. Week 48	1.4 %predicted normal Standard Deviation 7.98	-1.4 %predicted normal Standard Deviation 7.56

SECONDARY outcome

Timeframe: From Baseline to Weeks 4 and 24

Population: Some subjects did not have ECG obtained at some visits.

Assessment of the safety of MOL compared to placebo

Outcome measures

Outcome measures
Measure	Molgramostim n=81 Participants Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution	Placebo n=83 Participants Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution
Change in QT Interval Corrected by Fridericia (QTcF) Week 4	-2.51 msec Standard Deviation 10.543	1.59 msec Standard Deviation 10.127
Change in QT Interval Corrected by Fridericia (QTcF) Week 24	-1.89 msec Standard Deviation 13.190	-0.96 msec Standard Deviation 11.534

Adverse Events

Molgramostim

Serious events: 14 serious events

Other events: 69 other events

Deaths: 0 deaths

Placebo

Serious events: 20 serious events

Other events: 71 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Raymond D Pratt, MD FACP

Savara Inc.

Phone: 512 614 1848

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Molgramostim n=81 participants at risk Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution 79 subjects completed double-blind treatment and continued in the open-label extension. The open-label study is still ongoing.	Placebo n=83 participants at risk Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution 81 subjects completed double-blind treatment and enrolled in the open-label extension. The open-label study is still ongoing.
Respiratory, thoracic and mediastinal disorders Alveolar proteinosis	3.7% 3/81 • Number of events 3 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	10.8% 9/83 • Number of events 9 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Dyspnoea	2.5% 2/81 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Hypoxia	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	3.6% 3/83 • Number of events 3 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations COVID-19	2.5% 2/81 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	0.00% 0/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Pneumonia	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Mycobacterium avium complex infection	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	0.00% 0/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Brain abscess	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Bronchitis	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Ear and labyrinth disorders Sudden hearing loss	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	0.00% 0/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Injury, poisoning and procedural complications Clavicle fracture	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	0.00% 0/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Psychiatric disorders Delusion	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	0.00% 0/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Reproductive system and breast disorders Uterine polyp	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	0.00% 0/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Hypersensitivity pneumonitis	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Obstructive sleep apnea	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Eye disorders Cataract	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
General disorders Non-cardiac chest pain	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Metabolism and nutrition disorders Diabetes mellitus	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Nervous system disorders Intracranial mass	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Renal and urinary disorders Ureterolithiasis	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Skin and subcutaneous tissue disorders Angioedema	0.00% 0/81 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Influenza	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	0.00% 0/83 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Pneumonia	1.2% 1/81 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.

Measure	Molgramostim n=81 participants at risk Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks Molgramostim: Molgramostim 300 µg nebulizer solution 79 subjects completed double-blind treatment and continued in the open-label extension. The open-label study is still ongoing.	Placebo n=83 participants at risk Double-blind treatment with placebo nebulizer solution once daily for 48 weeks Placebo: Matching placebo nebulizer solution 81 subjects completed double-blind treatment and enrolled in the open-label extension. The open-label study is still ongoing.
Respiratory, thoracic and mediastinal disorders Alveolar proteinosis	2.5% 2/81 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	4.8% 4/83 • Number of events 4 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Vascular disorders Hypertension	4.9% 4/81 • Number of events 5 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	6.0% 5/83 • Number of events 5 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
General disorders Fatigue	2.5% 2/81 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	7.2% 6/83 • Number of events 8 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Upper respiratory tract infection	4.9% 4/81 • Number of events 5 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	3.6% 3/83 • Number of events 4 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Covid-19	19.8% 16/81 • Number of events 17 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	9.6% 8/83 • Number of events 8 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Cough	21.0% 17/81 • Number of events 30 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	21.7% 18/83 • Number of events 23 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Nasopharyngitis	13.6% 11/81 • Number of events 19 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	8.4% 7/83 • Number of events 9 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
General disorders Pyrexia	13.6% 11/81 • Number of events 16 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	10.8% 9/83 • Number of events 10 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Musculoskeletal and connective tissue disorders Arthralgia	11.1% 9/81 • Number of events 9 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	8.4% 7/83 • Number of events 7 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Gastrointestinal disorders Diarrhea	11.1% 9/81 • Number of events 10 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	2.4% 2/83 • Number of events 3 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Nervous system disorders Headache	11.1% 9/81 • Number of events 33 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	8.4% 7/83 • Number of events 8 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Musculoskeletal and connective tissue disorders Back pain	8.6% 7/81 • Number of events 7 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	3.6% 3/83 • Number of events 3 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Nervous system disorders Dizziness	8.6% 7/81 • Number of events 8 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	2.4% 2/83 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Dyspnoea	6.2% 5/81 • Number of events 6 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	3.6% 3/83 • Number of events 5 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
General disorders Non-cardiac chest pain	8.6% 7/81 • Number of events 8 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	6.0% 5/83 • Number of events 8 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Gastrointestinal disorders Nausea	7.4% 6/81 • Number of events 10 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	2.4% 2/83 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	7.4% 6/81 • Number of events 12 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	3.6% 3/83 • Number of events 3 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Gastrointestinal disorders Vomiting	7.4% 6/81 • Number of events 7 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Bronchitis	6.2% 5/81 • Number of events 5 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 1 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Pneumonia	4.9% 4/81 • Number of events 4 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.
Infections and infestations Urinary tract infection	4.9% 4/81 • Number of events 4 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.	1.2% 1/83 • Number of events 2 • Adverse events during double-blind treatment (48 Weeks) Adverse events only reported for the 48-week double-blind part of the study. The open label extension is currently ongoing and has not been analyzed. Results will be reported when the open-label study is complete.