Trial · NCT04544293
Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
NCT04544293, Phase 3, Active not recruiting, Savara Inc.; results first posted 2025-08-07
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP). Trials, record NCT04544293. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04544293
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
- NCT number
- NCT04544293
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 164 participants, actual
- Lead sponsor
- Savara Inc.
- Conditions
- Autoimmune Pulmonary Alveolar Proteinosis
- Interventions
- Molgramostim, Placebo, Molgramostim Open-label
- First posted
- 2020-09-10
- Last updated
- 2026-11-03
- Other registries' IDs
- eudract:2020-001263-85
- Results first posted
- 2025-08-07
Source ClinicalTrials.gov
Registered outcomes14
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Percent (%) Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) Adjusted for Hemoglobin Concentration to Week 24 | From Baseline to Week 24 | 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). |
| Secondary | Change From Baseline in Percent (%) Predicted DLCO Adjusted for Hemoglobin Concentration (%DLCOadj) to Week 48 | From Baseline to Week 48 | 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). Higher values indicate better respiratory gas exchange. |
| Secondary | Change From Baseline in St. Georges Respiratory Questionnaire (SGRQ) Total Score to Week 24 | 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. |
| Secondary | Change From Baseline in SGRQ Activity Component Score to Week 24 | 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 |
| Secondary | Change From Baseline in Exercise Capacity (EC), Expressed as Peak Metabolic Equivalents (METs) to Week 24 | 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. |
| Secondary | Change From Baseline in SGRQ Total Score to Week 48 | From Baseline to Week 48 | 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. |
| Secondary | Change From Baseline in SGRQ Activity From Baseline to Week 48 | From Baseline to Week 48 | 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 |
| Secondary | Change From Baseline in EC, Expressed as Peak METs to Week 48 | From Baseline to Week 48 | 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. |
| Secondary | Change From Baseline in Alveolar-arterial Oxygen Difference (A-aDO2) to Week 24 (All Subjects) | From Baseline to Week 24 | A-aDO2 was used as an additional measure of gas exchange. |
| Secondary | Number of Subjects With Serious and Non-serious Adverse Events | From Baseline until End of Double-blind treatment (Week 48) | Assessment of the safety of molgramostim compared to placebo |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Percent (%) Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) Adjusted for Hemoglobin Concentration to Week 24 | From Baseline to Week 24 | Full Analysis Set | % predicted DLCO adjusted for hemoglobin | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Percent (%) Predicted DLCO Adjusted for Hemoglobin Concentration (%DLCOadj) to Week 48 | From Baseline to Week 48 | Unit of Measure: % predicted DLCO adjust | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline in St. Georges Respiratory Questionnaire (SGRQ) Total Score to Week 24 | From Baseline to Week 24 | Change from baseline units on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline in SGRQ Activity Component Score to Week 24 | From Baseline to Week 24 | units on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline in Exercise Capacity (EC), Expressed as Peak Metabolic Equivalents (METs) to Week 24 | From Baseline to Week 24 | Change from baseline in METS | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline in SGRQ Total Score to Week 48 | From Baseline to Week 48 | Change from baseline units on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline in SGRQ Activity From Baseline to Week 48 | From Baseline to Week 48 | Change from baseline score on a scale | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline in EC, Expressed as Peak METs to Week 48 | From Baseline to Week 48 | Change from baseline in METS | LEAST_SQUARES_MEAN | |
| SECONDARY | Change From Baseline in Alveolar-arterial Oxygen Difference (A-aDO2) to Week 24 (All Subjects) | From Baseline to Week 24 | mmHg | LEAST_SQUARES_MEAN | |
| SECONDARY | Number of Subjects With Serious and Non-serious Adverse Events | From Baseline until End of Double-blind treatment (Week 48) | participants | NUMBER |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group38
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Percent (%) Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) Adjusted for Hemoglobin Concentration to Week 24 | Molgramostim | 9.8 | 95% Confidence Interval 7.3 to 12.3 | % predicted DLCO adjusted for hemoglobin | |
| Change From Baseline in Percent (%) Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) Adjusted for Hemoglobin Concentration to Week 24 | Placebo | 3.8 | 95% Confidence Interval 1.4 to 6.3 | % predicted DLCO adjusted for hemoglobin | |
| Change From Baseline in Percent (%) Predicted DLCO Adjusted for Hemoglobin Concentration (%DLCOadj) to Week 48 | Molgramostim | 11.6 | 95% Confidence Interval 8.7 to 14.5 | Unit of Measure: % predicted DLCO adjust | |
| Change From Baseline in Percent (%) Predicted DLCO Adjusted for Hemoglobin Concentration (%DLCOadj) to Week 48 | Placebo | 4.7 | 95% Confidence Interval 1.8 to 7.6 | Unit of Measure: % predicted DLCO adjust | |
| Change From Baseline in St. Georges Respiratory Questionnaire (SGRQ) Total Score to Week 24 | Molgramostim | -11.5 | 95% Confidence Interval -15.01 to -7.98 | Change from baseline units on a scale | |
| Change From Baseline in St. Georges Respiratory Questionnaire (SGRQ) Total Score to Week 24 | Placebo | -4.9 | 95% Confidence Interval -8.28 to -1.52 | Change from baseline units on a scale | |
| Change From Baseline in SGRQ Activity Component Score to Week 24 | Molgramostim | -13.03 | 95% Confidence Interval -17.58 to -8.49 | units on a scale | |
| Change From Baseline in SGRQ Activity Component Score to Week 24 | Placebo | -5.22 | 95% Confidence Interval -9.76 to -0.69 | units on a scale | |
| Change From Baseline in Exercise Capacity (EC), Expressed as Peak Metabolic Equivalents (METs) to Week 24 | Molgramostim | 1.11 | 95% Confidence Interval 0.76 to 1.46 | Change from baseline in METS | |
| Change From Baseline in Exercise Capacity (EC), Expressed as Peak Metabolic Equivalents (METs) to Week 24 | Placebo | 0.70 | 95% Confidence Interval 0.34 to 1.05 | Change from baseline in METS |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double-blind | STARTED | Molgramostim | 81 | |
| Double-blind | STARTED | Placebo | 83 | |
| Double-blind | COMPLETED | Molgramostim | 79 | |
| Double-blind | COMPLETED | Placebo | 80 | |
| Double-blind | NOT COMPLETED | Molgramostim | 2 | |
| Double-blind | NOT COMPLETED | Placebo | 3 | |
| Open-Label Extension | STARTED | Molgramostim | 79 | |
| Open-Label Extension | STARTED | Placebo | 81 | |
| Open-Label Extension | COMPLETED | Molgramostim | 0 | |
| Open-Label Extension | COMPLETED | Placebo | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events90
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 | Covid-19 | Infections and infestations | Molgramostim | 16 | 81 | 17 |
| other | Covid-19 | Infections and infestations | Placebo | 8 | 83 | 8 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Molgramostim | 17 | 81 | 30 |
| other | Cough | Respiratory, thoracic and mediastinal disorders | Placebo | 18 | 83 | 23 |
| other | Nasopharyngitis | Infections and infestations | Molgramostim | 11 | 81 | 19 |
| other | Nasopharyngitis | Infections and infestations | Placebo | 7 | 83 | 9 |
| other | Pyrexia | General disorders | Molgramostim | 11 | 81 | 16 |
| other | Pyrexia | General disorders | Placebo | 9 | 83 | 10 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Molgramostim | 9 | 81 | 9 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Placebo | 7 | 83 | 7 |
1 to 10 of 90Source ClinicalTrials.gov
See all 90 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Molgramostim | 0 | 81 |
| Total, all-cause mortality | Placebo | 0 | 83 |
| Total, other adverse events | Molgramostim | 69 | 81 |
| Total, other adverse events | Placebo | 71 | 83 |
| Total, serious adverse events | Molgramostim | 14 | 81 |
| Total, serious adverse events | Placebo | 20 | 83 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40834301 | DERIVED |
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Molgramostim vs Placebo: deaths from any cause | Trial comparisons |
| Molgramostim vs Placebo: serious adverse events | Trial comparisons |
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.
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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04544293
GET https://conductscience.org/api/trials/v1/NCT04544293/results?page=2