Trial · NCT02702180

Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis

NCT02702180, Phase 2, Completed, Savara Inc.; results first posted 2022-04-15

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis. Trials, record NCT02702180. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02702180

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Efficacy and Safety of Inhaled Molgramostim (rhGM-CSF) in Autoimmune Pulmonary Alveolar Proteinosis
NCT number: NCT02702180
Status: Completed
Phases: Phase 2
Enrollment: 139 participants, actual
Lead sponsor: Savara Inc.
Conditions: Autoimmune Pulmonary Alveolar Proteinosis
Interventions: Molgramostim, Placebo, PARI eFlow nebulizer system
First posted: 2016-03-08
Last updated: 2023-04-12
Other registries' IDs: eudract:2015-003878-33
Results first posted: 2022-04-15

Source ClinicalTrials.gov

Registered outcomes9

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment	From baseline to 24 weeks	Measurement of (A-a)DO2 was done by blood gas analysis. An arterial blood sample was collected in the supine position, after resting for at least 10 minutes (or longer if required to achieve stable oxygen saturation). The sample was analyzed for arterial oxygen tension (PaO2) and partial pressure of carbon dioxide (PaCO2). The calculation of (A-a)DO2 was done centrally by using a formula described in the protocol.
Secondary	Change From Baseline in 6-minute Walking Distance (6MWD) After 24 Weeks of Treatment	From baseline to 24 weeks	The 6MWD was assessed by the use of 6-minute walking test (6MWT). The 6MWT was performed in accordance with the 2014 ATS/ERS guideline "field walking tests in chronic respiratory disease" (Holland et al. 2014) by technicians with documented training and experience. Where possible, the test was conducted with the participant breathing ambient atmospheric air. If the participant required oxygen supplementation at rest, a titration procedure was carried out as part of the 6MWT at screening in order to determine the oxygen flow rate required for the participant to complete the test. This flow rate was to be used during subsequent tests, if possible.
Secondary	Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score After 24 Weeks of Treatment	From baseline to 24 weeks	The SGRQ is designed to measure health impairment in patients with asthma and chronic obstructive pulmonary disease (COPD). It consists of two parts, where Part 1 covers the participants' recollection of their symptoms over the preceding period (1 month recall used in this trial, Symptoms component) and Part 2 addresses the participants' current state in terms of disturbances to their daily physical activity (Activity component) and a wide range of disturbances of psycho-social function (Impact component). A total score as well as individual component scores were calculated. Scores (total and component) range from 0 to 100, with higher scores indicating more limitations.
Secondary	Number of Whole Lung Lavage During 24 Weeks of Treatment	From baseline to 24 weeks	In all versions of the protocol (no participants were recruited under version 1.0), whole lung lavage (single lung or both lungs) was applied as rescue therapy. In protocol version 2.0, the criterion for performing whole lung lavage was an increase in (A-a)DO2 by more than 10 mmHg/1.33 kilopascal compared to baseline. In protocol version 3.0 onwards, the criterion for performing whole lung lavage was clinical worsening of aPAP based on symptoms, reduced exercise capacity and/or findings of hypoxemia or desaturation according to the investigator's judgement. In protocol versions 2.0 and 3.0, participants undergoing whole lung lavage during the double-blind period were to discontinue double-blind treatment, encouraged to continue to follow the same visit schedule and, if required, receive further whole lung lavage at the investigator's discretion. In protocol version 4.0 and 5.0, participants undergoing whole lung lavage were to continue double-blind treatment.
Secondary	Number of Adverse Events (AEs) During 24 Weeks of Treatment	From baseline to 24 weeks	Treatment-emergent adverse events (AEs) were assessed on or after the first dose of trial drug to 28 days after last dose. Information about AEs was collected by the investigator by a non-leading question such as "have you experienced any new health problems or worsening of existing conditions" and by reporting events directly observed or spontaneously volunteered by participants (e.g., in the Subject Diary Card, where subjects were asked to record any AEs and answer questions regarding lung toxicity and known systemic effects). Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns. All AEs were assessed by the investigator for severity (mild, moderate, severe), outcome (recovered, not recovered, recovered with sequelae, fatal, unknown) and causality (unlikely, possible, probable, not applicable) according to current regulatory standards.
Secondary	Number of Serious Adverse Events (SAEs) During 24 Weeks of Treatment	From baseline to 24 weeks	SAEs are defined as any untoward medicinal occurrence or effect that at any dose: * Results in death * Is life-threatening * Requires hospitalisation or prolongation of existing hospitalisation * Results in persistent or significant disability or incapacity * Is a congenital anomaly or birth defect * May jeopardise the subject or may require intervention to prevent one or more of the other outcomes listed above (Important Medical Events)
Secondary	Number of Adverse Drug Reactions (ADRs) During 24 Weeks of Treatment	From baseline to 24 weeks	All AEs judged by either the reporting investigator or the sponsor as having a reasonable causal relationship to a medicinal product qualify as ADRs. The expression reasonable causal relationship means that a causal relationship between a medicinal product and an AE is at least a reasonable possibility i.e. the relationship cannot be ruled out.
Secondary	Number of Severe AEs During 24 Weeks of Treatment	From baseline to 24 weeks	All AEs were assessed by the investigator for severity (mild, moderate, severe) according to current regulatory standards: * Mild: The AE is easily tolerated and does not interfere with daily activity. * Moderate: The AE interferes with daily activity, but the subject is still able to function. Medical intervention may be considered. * Severe: The AE is incapacitating and requires medical intervention.
Secondary	Number of Participants With at Least 1 AE Leading to Treatment Discontinuation During 24 Weeks of Treatment	From baseline to 24 weeks	Participants could be discontinued from treatment and assessments at any time, if deemed necessary by the investigator. Potential reasons for discontinuation of treatment included e.g. unacceptable AE.

Source ClinicalTrials.gov

Outcomes9

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment	From baseline to 24 weeks	Full analysis set (FAS): all randomized participants, analyzed according to randomized treatment. Not all participants in the FAS had blood gas analysis done at Week 24, therefore the overall number of participants analyzed is less than the total number of randomized participants.	mmHg	MEAN
SECONDARY	Change From Baseline in 6-minute Walking Distance (6MWD) After 24 Weeks of Treatment	From baseline to 24 weeks	FAS. Not all participants in the FAS had 6MWT done at Week 24, therefore the overall number of participants analyzed is less than the total number of randomized participants.	meters	MEAN
SECONDARY	Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score After 24 Weeks of Treatment	From baseline to 24 weeks	FAS. Not all participants in the FAS had SGRQ assessed at Week 24, therefore the overall number of participants analyzed is less than the total number of randomized participants.	score on a scale	MEAN
SECONDARY	Number of Whole Lung Lavage During 24 Weeks of Treatment	From baseline to 24 weeks	FAS.	lavages	NUMBER
SECONDARY	Number of Adverse Events (AEs) During 24 Weeks of Treatment	From baseline to 24 weeks	Safety analysis set: all randomized participants who received at least one dose of the trial drug, analyzed according to actual treatment.	events	NUMBER
SECONDARY	Number of Serious Adverse Events (SAEs) During 24 Weeks of Treatment	From baseline to 24 weeks	Safety analysis set	events	NUMBER
SECONDARY	Number of Adverse Drug Reactions (ADRs) During 24 Weeks of Treatment	From baseline to 24 weeks	Safety analysis set	events	NUMBER
SECONDARY	Number of Severe AEs During 24 Weeks of Treatment	From baseline to 24 weeks	Safety analysis set	events	NUMBER
SECONDARY	Number of Participants With at Least 1 AE Leading to Treatment Discontinuation During 24 Weeks of Treatment	From baseline to 24 weeks	Safety analysis set	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group27

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment	Molgramostim Once Daily	28.6	Standard Deviation 21.95	mmHg
Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment	Molgramostim Intermittent	29.3	Standard Deviation 17.96	mmHg
Absolute Change From Baseline of Alveolar-arterial Oxygen Concentration (A-a(DO2)) After 24 Weeks of Treatment	Placebo	32.0	Standard Deviation 20.90	mmHg
Change From Baseline in 6-minute Walking Distance (6MWD) After 24 Weeks of Treatment	Molgramostim Once Daily	39.6	Standard Deviation 95.89	meters
Change From Baseline in 6-minute Walking Distance (6MWD) After 24 Weeks of Treatment	Molgramostim Intermittent	11.3	Standard Deviation 81.94	meters
Change From Baseline in 6-minute Walking Distance (6MWD) After 24 Weeks of Treatment	Placebo	6.0	Standard Deviation 97.17	meters
Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score After 24 Weeks of Treatment	Molgramostim Once Daily	-12.3	Standard Deviation 14.30	score on a scale
Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score After 24 Weeks of Treatment	Molgramostim Intermittent	-12.0	Standard Deviation 15.12	score on a scale
Change From Baseline in St. George's Respiratory Questionnaire (SGRQ) Total Score After 24 Weeks of Treatment	Placebo	-4.7	Standard Deviation 12.83	score on a scale
Number of Whole Lung Lavage During 24 Weeks of Treatment	Molgramostim Once Daily	9		lavages

1 to 10 of 27Source ClinicalTrials.gov

See all 27 results by group

Participant flow18

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Double-blind Treatment Period	STARTED	Molgramostim Once Daily	46
Double-blind Treatment Period	STARTED	Molgramostim Intermittent	45
Double-blind Treatment Period	STARTED	Placebo	47
Double-blind Treatment Period	COMPLETED	Molgramostim Once Daily	45
Double-blind Treatment Period	COMPLETED	Molgramostim Intermittent	44
Double-blind Treatment Period	COMPLETED	Placebo	43
Double-blind Treatment Period	NOT COMPLETED	Molgramostim Once Daily	1
Double-blind Treatment Period	NOT COMPLETED	Molgramostim Intermittent	1
Double-blind Treatment Period	NOT COMPLETED	Placebo	4
Open-label Treatment Period	STARTED	Molgramostim Once Daily	45

1 to 10 of 18Source ClinicalTrials.gov

See all 18 participant flow

Adverse events176

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Cough	Respiratory, thoracic and mediastinal disorders	Double-blind Molgramostim Once Daily	15	46	22
other	Cough	Respiratory, thoracic and mediastinal disorders	Double-blind Molgramostim Intermittent	12	45	18
other	Cough	Respiratory, thoracic and mediastinal disorders	Double-blind Placebo	11	47	12
other	Cough	Respiratory, thoracic and mediastinal disorders	Open-label Molgramostim Intermittent	11	130	14
other	Nasopharyngitis	Infections and infestations	Double-blind Molgramostim Once Daily	7	46	10
other	Nasopharyngitis	Infections and infestations	Double-blind Molgramostim Intermittent	10	45	12
other	Nasopharyngitis	Infections and infestations	Double-blind Placebo	6	47	6
other	Nasopharyngitis	Infections and infestations	Open-label Molgramostim Intermittent	24	130	33
other	Headache	Nervous system disorders	Double-blind Molgramostim Once Daily	6	46	9
other	Headache	Nervous system disorders	Double-blind Molgramostim Intermittent	7	45	9

1 to 10 of 176Source ClinicalTrials.gov

See all 176 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Double-blind Molgramostim Once Daily	0	46
Total, all-cause mortality	Double-blind Molgramostim Intermittent	0	45
Total, all-cause mortality	Double-blind Placebo	0	47
Total, all-cause mortality	Open-label Molgramostim Intermittent	0	130
Total, other adverse events	Double-blind Molgramostim Once Daily	39	46
Total, other adverse events	Double-blind Molgramostim Intermittent	41	45
Total, other adverse events	Double-blind Placebo	41	47
Total, other adverse events	Open-label Molgramostim Intermittent	87	130
Total, serious adverse events	Double-blind Molgramostim Once Daily	8	46
Total, serious adverse events	Double-blind Molgramostim Intermittent	5	45

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
32897035	BACKGROUND

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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