Ignite Creation Date:
2025-12-25 @ 12:56 AM
Ignite Modification Date:
2025-12-25 @ 11:11 PM
Study NCT ID:
NCT04544293
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-08-07
First Post:
2020-09-03
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Russia'], 'submissionTracking': {'firstMcpInfo': {'postDateStruct': {'date': '2025-05-30', 'type': 'ACTUAL'}}}}, 'conditionBrowseModule': {'meshes': [{'id': 'C567049', 'term': 'Pulmonary Alveolar Proteinosis, Acquired'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C082430', 'term': 'molgramostim'}, {'id': 'D003115', 'term': 'Colony-Stimulating Factors'}, {'id': 'C082856', 'term': 'regramostim'}], 'ancestors': [{'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'ray.pratt@savarapharma.com', 'phone': '512 614 1848', 'title': 'Raymond D Pratt, MD FACP', 'organization': 'Savara Inc.'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Adverse events during double-blind treatment (48 Weeks)', 'description': '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.', 'eventGroups': [{'id': 'EG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution\n\n79 subjects completed double-blind treatment and continued in the open-label extension. The open-label study is still ongoing.', 'otherNumAtRisk': 81, 'deathsNumAtRisk': 81, 'otherNumAffected': 69, 'seriousNumAtRisk': 81, 'deathsNumAffected': 0, 'seriousNumAffected': 14}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution\n\n81 subjects completed double-blind treatment and enrolled in the open-label extension. The open-label study is still ongoing.', 'otherNumAtRisk': 83, 'deathsNumAtRisk': 83, 'otherNumAffected': 71, 'seriousNumAtRisk': 83, 'deathsNumAffected': 0, 'seriousNumAffected': 20}], 'otherEvents': [{'term': 'Covid-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 17, 'numAffected': 16}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 8, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 30, 'numAffected': 17}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 23, 'numAffected': 18}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 19, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 9, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 16, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 10, 'numAffected': 9}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 9, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 7, 'numAffected': 7}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Diarrhea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 10, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 33, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 8, 'numAffected': 7}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 8, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 6, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 8, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 8, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 10, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 12, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 7, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 5, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Alveolar proteinosis', 'notes': 'Worsening of aPAP', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 4, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 5, 'numAffected': 5}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 8, 'numAffected': 6}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 5, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 4, 'numAffected': 3}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}], 'seriousEvents': [{'term': 'Alveolar proteinosis', 'notes': 'Worsening of aPAP', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 9, 'numAffected': 9}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Hypoxia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Mycobacterium avium complex infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Brain abscess', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Sudden hearing loss', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Clavicle fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Delusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numAffected': 0}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Uterine polyp', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Reproductive system and breast disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Hypersensitivity pneumonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Obstructive sleep apnea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Pulmonary embolism', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Cataract', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Non-cardiac chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Intracranial mass', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Ureterolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Angioedema', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 81, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 83, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 27.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Percent (%) Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) Adjusted for Hemoglobin Concentration to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '9.8', 'groupId': 'OG000', 'lowerLimit': '7.3', 'upperLimit': '12.3'}, {'value': '3.8', 'groupId': 'OG001', 'lowerLimit': '1.4', 'upperLimit': '6.3'}]}]}], 'analyses': [{'pValue': '0.0007', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '6.0', 'ciLowerLimit': '2.5', 'ciUpperLimit': '9.4', 'estimateComment': 'Difference from placebo', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Modelled using MMRM adjusted for baseline % predicted DLCOadj, treatment, visit, region, severity stratification and treatment-visit interaction.'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 24', 'description': '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).', 'unitOfMeasure': '% predicted DLCO adjusted for hemoglobin', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Percent (%) Predicted DLCO Adjusted for Hemoglobin Concentration (%DLCOadj) to Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '11.6', 'groupId': 'OG000', 'lowerLimit': '8.7', 'upperLimit': '14.5'}, {'value': '4.7', 'groupId': 'OG001', 'lowerLimit': '1.8', 'upperLimit': '7.6'}]}]}], 'analyses': [{'pValue': '0.0008', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '6.9', 'ciLowerLimit': '2.9', 'ciUpperLimit': '10.9', 'pValueComment': 'Modelled using MMRM adjusted for baseline % predicted DLCOadj, treatment, visit, region, severity stratification and treatment-visit interaction.', 'estimateComment': 'Difference from Placebo', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 48', 'description': '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.', 'unitOfMeasure': 'Unit of Measure: % predicted DLCO adjust', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in St. Georges Respiratory Questionnaire (SGRQ) Total Score to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '-11.5', 'groupId': 'OG000', 'lowerLimit': '-15.01', 'upperLimit': '-7.98'}, {'value': '-4.9', 'groupId': 'OG001', 'lowerLimit': '-8.28', 'upperLimit': '-1.52'}]}]}], 'analyses': [{'pValue': '0.0072', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-6.59', 'ciLowerLimit': '-11.40', 'ciUpperLimit': '-1.79', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 24', 'description': "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.", 'unitOfMeasure': 'Change from baseline units on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in SGRQ Activity Component Score to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '-13.03', 'groupId': 'OG000', 'lowerLimit': '-17.58', 'upperLimit': '-8.49'}, {'value': '-5.22', 'groupId': 'OG001', 'lowerLimit': '-9.76', 'upperLimit': '-0.69'}]}]}], 'analyses': [{'pValue': '0.0149', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-7.81', 'ciLowerLimit': '-14.10', 'ciUpperLimit': '-1.52', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 24', 'description': "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", 'unitOfMeasure': 'units on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Exercise Capacity (EC), Expressed as Peak Metabolic Equivalents (METs) to Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '1.11', 'groupId': 'OG000', 'lowerLimit': '0.76', 'upperLimit': '1.46'}, {'value': '0.70', 'groupId': 'OG001', 'lowerLimit': '0.34', 'upperLimit': '1.05'}]}]}], 'analyses': [{'pValue': '0.0845', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.41', 'ciLowerLimit': '-0.06', 'ciUpperLimit': '0.89', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 24', 'description': '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.', 'unitOfMeasure': 'Change from baseline in METS', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in SGRQ Total Score to Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '-10.72', 'groupId': 'OG000', 'lowerLimit': '-15.01', 'upperLimit': '-6.44'}, {'value': '-5.85', 'groupId': 'OG001', 'lowerLimit': '-9.96', 'upperLimit': '-1.74'}]}]}], 'analyses': [{'pValue': '0.1046', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-4.87', 'ciLowerLimit': '-10.76', 'ciUpperLimit': '1.01', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 48', 'description': "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.", 'unitOfMeasure': 'Change from baseline units on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in SGRQ Activity From Baseline to Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '-13.39', 'groupId': 'OG000', 'lowerLimit': '-18.87', 'upperLimit': '-7.91'}, {'value': '-7.40', 'groupId': 'OG001', 'lowerLimit': '-12.72', 'upperLimit': '-2.09'}]}]}], 'analyses': [{'pValue': '0.1216', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-5.99', 'ciLowerLimit': '-13.57', 'ciUpperLimit': '1.59', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 48', 'description': "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", 'unitOfMeasure': 'Change from baseline score on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in EC, Expressed as Peak METs to Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '1.13', 'groupId': 'OG000', 'lowerLimit': '0.77', 'upperLimit': '1.49'}, {'value': '0.58', 'groupId': 'OG001', 'lowerLimit': '0.22', 'upperLimit': '0.93'}]}]}], 'analyses': [{'pValue': '0.0234', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.55', 'ciLowerLimit': '0.07', 'ciUpperLimit': '1.03', 'estimateComment': 'Change from baseline compared to placebo.', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 48', 'description': '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.', 'unitOfMeasure': 'Change from baseline in METS', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Alveolar-arterial Oxygen Difference (A-aDO2) to Week 24 (All Subjects)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'categories': [{'measurements': [{'value': '-7.97', 'groupId': 'OG000', 'lowerLimit': '-11.62', 'upperLimit': '-4.32'}, {'value': '-3.96', 'groupId': 'OG001', 'lowerLimit': '-7.48', 'upperLimit': '-0.44'}]}]}], 'analyses': [{'pValue': '0.1043', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-4.01', 'ciLowerLimit': '-8.84', 'ciUpperLimit': '0.83', 'statisticalMethod': 'Mixed Models Analysis', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 24', 'description': 'A-aDO2 was used as an additional measure of gas exchange.', 'unitOfMeasure': 'mmHg', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Number of Subjects With Serious and Non-serious Adverse Events', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'title': 'Any TEAE', 'categories': [{'measurements': [{'value': '69', 'groupId': 'OG000'}, {'value': '71', 'groupId': 'OG001'}]}]}, {'title': 'Any TESAE', 'categories': [{'measurements': [{'value': '14', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From Baseline until End of Double-blind treatment (Week 48)', 'description': 'Assessment of the safety of molgramostim compared to placebo', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': "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", 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '80', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '28', 'groupId': 'OG000'}, {'value': '30', 'groupId': 'OG001'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '80', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '39', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From Baseline until End of Double-blind treatment Week-48', 'description': '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).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'One subject in the molgramostim group had missing antiGM-CSF titers.'}, {'type': 'SECONDARY', 'title': 'Changes in Forced Vital Capacity (FVC) %Predicted Normal', 'denoms': [{'units': 'Participants', 'counts': [{'value': '78', 'groupId': 'OG000'}, {'value': '81', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'title': 'Week 24', 'categories': [{'measurements': [{'value': '3.2', 'spread': '6.20', 'groupId': 'OG000'}, {'value': '0.9', 'spread': '5.74', 'groupId': 'OG001'}]}]}, {'title': 'Week 48', 'categories': [{'measurements': [{'value': '3.2', 'spread': '7.92', 'groupId': 'OG000'}, {'value': '-0.1', 'spread': '8.27', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Weeks 24 and 48', 'description': '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.', 'unitOfMeasure': '% FVC predicted', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Changes in Forced Expiratory Volume in One Second (FEV1) % Predicted Normal.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '78', 'groupId': 'OG000'}, {'value': '81', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'title': 'Week 24', 'categories': [{'measurements': [{'value': '1.2', 'spread': '7.08', 'groupId': 'OG000'}, {'value': '-0.3', 'spread': '5.35', 'groupId': 'OG001'}]}]}, {'title': 'Week 48', 'categories': [{'measurements': [{'value': '1.4', 'spread': '7.98', 'groupId': 'OG000'}, {'value': '-1.4', 'spread': '7.56', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Weeks 24 and 48', 'description': 'FEV1 is the volume of air expired in 1 second in liters (L). Higher values indicate better respiratory function.', 'unitOfMeasure': '%predicted normal', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Change in QT Interval Corrected by Fridericia (QTcF)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'classes': [{'title': 'Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '80', 'groupId': 'OG000'}, {'value': '83', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-2.51', 'spread': '10.543', 'groupId': 'OG000'}, {'value': '1.59', 'spread': '10.127', 'groupId': 'OG001'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '79', 'groupId': 'OG000'}, {'value': '80', 'groupId': 'OG001'}]}], 'categories': [{'measurements': [{'value': '-1.89', 'spread': '13.190', 'groupId': 'OG000'}, {'value': '-0.96', 'spread': '11.534', 'groupId': 'OG001'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'From Baseline to Weeks 4 and 24', 'description': 'Assessment of the safety of MOL compared to placebo', 'unitOfMeasure': 'msec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Some subjects did not have ECG obtained at some visits.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}], 'periods': [{'title': 'Double-blind', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '81'}, {'groupId': 'FG001', 'numSubjects': '83'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '79'}, {'comment': 'one subject in Placebo discontinued blinded IMP but completed all study visits and assessments', 'groupId': 'FG001', 'numSubjects': '80'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '3'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Pregnancy', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}, {'title': 'Open-Label Extension', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '79'}, {'comment': 'One subject discontinued IMP in the double-blind period and completed all study visits then entered the OLE', 'groupId': 'FG001', 'numSubjects': '81'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '79'}, {'groupId': 'FG001', 'numSubjects': '81'}]}], 'dropWithdraws': [{'type': 'Study ongoing', 'reasons': [{'groupId': 'FG000', 'numSubjects': '79'}, {'groupId': 'FG001', 'numSubjects': '81'}]}]}], 'recruitmentDetails': 'Outpatient Medical Clinics 19 May 2021 -16 June 2023', 'preAssignmentDetails': '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.\n\nTo 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%.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '81', 'groupId': 'BG000'}, {'value': '83', 'groupId': 'BG001'}, {'value': '164', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily for 48 weeks\n\nMolgramostim: Molgramostim 300 µg nebulizer solution'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Double-blind treatment with placebo nebulizer solution once daily for 48 weeks\n\nPlacebo: Matching placebo nebulizer solution'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '50.8', 'spread': '13.03', 'groupId': 'BG000'}, {'value': '48.4', 'spread': '12.69', 'groupId': 'BG001'}, {'value': '49.6', 'spread': '12.87', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '37', 'groupId': 'BG000'}, {'value': '29', 'groupId': 'BG001'}, {'value': '66', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '44', 'groupId': 'BG000'}, {'value': '54', 'groupId': 'BG001'}, {'value': '98', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '36', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '73', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '38', 'groupId': 'BG000'}, {'value': '40', 'groupId': 'BG001'}, {'value': '78', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '8', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'Romania', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '6', 'groupId': 'BG002'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '11', 'groupId': 'BG000'}, {'value': '12', 'groupId': 'BG001'}, {'value': '23', 'groupId': 'BG002'}]}]}, {'title': 'Japan', 'categories': [{'measurements': [{'value': '29', 'groupId': 'BG000'}, {'value': '24', 'groupId': 'BG001'}, {'value': '53', 'groupId': 'BG002'}]}]}, {'title': 'United Kingdom', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}, {'title': 'Portugal', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Spain', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Canada', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}]}, {'title': 'South Korea', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}]}, {'title': 'Turkey', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '10', 'groupId': 'BG001'}, {'value': '18', 'groupId': 'BG002'}]}]}, {'title': 'Belgium', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'Ireland', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG002'}]}]}, {'title': 'Poland', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}]}, {'title': 'Italy', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}]}, {'title': 'Australia', 'categories': [{'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}]}, {'title': 'France', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}]}, {'title': 'Germany', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}, {'title': '%DLCOadj at Baseline', 'classes': [{'categories': [{'measurements': [{'value': '54.0', 'groupId': 'BG000', 'lowerLimit': '25', 'upperLimit': '72'}, {'value': '55.0', 'groupId': 'BG001', 'lowerLimit': '28', 'upperLimit': '71'}, {'value': '55', 'groupId': 'BG002', 'lowerLimit': '25', 'upperLimit': '72'}]}]}], 'paramType': 'MEDIAN', 'description': 'Percent predicted diffusion capacity for carbon monoxide adjusted for hemoglobin concentration.', 'unitOfMeasure': '%', 'dispersionType': 'FULL_RANGE'}, {'title': '%DLCOadj at Randomization <=50%', 'classes': [{'categories': [{'measurements': [{'value': '31', 'groupId': 'BG000'}, {'value': '32', 'groupId': 'BG001'}, {'value': '63', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Percent predicted diffusion capacity for carbon monoxide adjusted for hemoglobin concentration less than or equal to 50% predicted.', 'unitOfMeasure': 'Participants'}, {'title': '%DLCOadj at Randomization >50%', 'classes': [{'categories': [{'measurements': [{'value': '50', 'groupId': 'BG000'}, {'value': '51', 'groupId': 'BG001'}, {'value': '101', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'Percent predicted diffusion capacity for carbon monoxide adjusted for hemoglobin concentration greater than 50%', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'One subject in the placebo group stopped randomized drug but continued in the study to complete all visits and assessments'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2024-10-09', 'size': 4514848, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2025-04-25T14:17', 'hasProtocol': True}, {'date': '2024-04-24', 'size': 4816368, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2025-04-25T14:20', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Subject will be randomized 1:1 to treatment with inhaled molgramostim or placebo'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 164}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-05-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2027-05-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-18', 'studyFirstSubmitDate': '2020-09-03', 'resultsFirstSubmitDate': '2025-05-12', 'studyFirstSubmitQcDate': '2020-09-03', 'lastUpdatePostDateStruct': {'date': '2025-08-07', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2025-07-18', 'studyFirstPostDateStruct': {'date': '2020-09-10', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-08-07', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-11-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Percent (%) Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) Adjusted for Hemoglobin Concentration to Week 24', 'timeFrame': 'From Baseline to Week 24', 'description': '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).'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Percent (%) Predicted DLCO Adjusted for Hemoglobin Concentration (%DLCOadj) to Week 48', 'timeFrame': 'From Baseline to Week 48', 'description': '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.'}, {'measure': 'Change From Baseline in St. Georges Respiratory Questionnaire (SGRQ) Total Score to Week 24', 'timeFrame': 'From Baseline to Week 24', 'description': "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."}, {'measure': 'Change From Baseline in SGRQ Activity Component Score to Week 24', 'timeFrame': 'From Baseline to Week 24', 'description': "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"}, {'measure': 'Change From Baseline in Exercise Capacity (EC), Expressed as Peak Metabolic Equivalents (METs) to Week 24', 'timeFrame': 'From Baseline to Week 24', 'description': '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.'}, {'measure': 'Change From Baseline in SGRQ Total Score to Week 48', 'timeFrame': 'From Baseline to Week 48', 'description': "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."}, {'measure': 'Change From Baseline in SGRQ Activity From Baseline to Week 48', 'timeFrame': 'From Baseline to Week 48', 'description': "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"}, {'measure': 'Change From Baseline in EC, Expressed as Peak METs to Week 48', 'timeFrame': 'From Baseline to Week 48', 'description': '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.'}, {'measure': 'Change From Baseline in Alveolar-arterial Oxygen Difference (A-aDO2) to Week 24 (All Subjects)', 'timeFrame': 'From Baseline to Week 24', 'description': 'A-aDO2 was used as an additional measure of gas exchange.'}, {'measure': 'Number of Subjects With Serious and Non-serious Adverse Events', 'timeFrame': 'From Baseline until End of Double-blind treatment (Week 48)', 'description': 'Assessment of the safety of molgramostim compared to placebo'}, {'measure': "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", 'timeFrame': 'From Baseline until End of Double-blind treatment Week-48', 'description': '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).'}, {'measure': 'Changes in Forced Vital Capacity (FVC) %Predicted Normal', 'timeFrame': 'From Baseline to Weeks 24 and 48', 'description': '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.'}, {'measure': 'Changes in Forced Expiratory Volume in One Second (FEV1) % Predicted Normal.', 'timeFrame': 'From Baseline to Weeks 24 and 48', 'description': 'FEV1 is the volume of air expired in 1 second in liters (L). Higher values indicate better respiratory function.'}, {'measure': 'Change in QT Interval Corrected by Fridericia (QTcF)', 'timeFrame': 'From Baseline to Weeks 4 and 24', 'description': 'Assessment of the safety of MOL compared to placebo'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['aPAP'], 'conditions': ['Autoimmune Pulmonary Alveolar Proteinosis']}, 'referencesModule': {'references': [{'pmid': '40834301', 'type': 'DERIVED', 'citation': 'Trapnell BC, Inoue Y, Bonella F, Wang T, McCarthy C, Arai T, Akasaka K, Mariani F, Mogulkoc N, Song JW, Baba T, Jouneau S, Numakura T, Ocal N, Mihaltan F, Ataya A, Bendstrup E, Campo I, Carey B, Arena R, Robinson B, Fleming R, Wasfi Y, Pratt R; IMPALA-2 Trial Investigators. Phase 3 Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis. N Engl J Med. 2025 Aug 21;393(8):764-773. doi: 10.1056/NEJMoa2410542.'}]}, 'descriptionModule': {'briefSummary': '160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim (MOL) or placebo (PBO) for 48 weeks. Subjects completing the 48-week placebo-controlled period will receive open-label treatment with once daily inhaled molgramostim for 96 weeks.', 'detailedDescription': 'This is an interventional, randomized, double-blind, 2-arm, parallel groups, placebo-controlled, multi-center, phase 3 trial in adult subjects who are diagnosed with aPAP.\n\nAn aPAP diagnosis should be confirmed by a Granulocyte-macrophage colony stimulating factor (GM-CSF) auto-antibody test result, and history of PAP based on either high resolution computed tomography, lung biopsy, or bronchoalveolar lavage cytology, should be available.\n\nThe trial consists of a 6-week screening period, a 48-week randomized, double-blind treatment period, a 96-week open-label treatment period, and a conditional 4-week safety follow-up period. The maximum treatment duration will be 145 weeks and the maximum trial duration will be 156 weeks. During the trial, whole lung lavage will be allowed as rescue treatment in case of worsening of aPAP.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Subject must be ≥18 years of age, at the time of signing the informed consent (≥20 in Japan).\n2. A serum anti-GM-CSF autoantibody test result confirming autoimmune PAP.\n3. History of PAP, based on examination of a lung biopsy, bronchoalveolar lavage (BAL) cytology, or a high-resolution computed tomogram (HRCT) of the chest.\n4. A diffusing capacity for carbon monoxide of 70% predicted or lower adjusted for hemoglobin (%DLCOadj) at the screening and baseline visits.\n5. Change in %DLCO adj of \\<15% points during the screening period.\n6. Demonstrated functional impairment in the treadmill exercise test (defined as a peak metabolic equivalent (MET) ≤8).\n7. Willing and able to come off supplemental oxygen use prior to and during the treadmill exercise test, the DLCO assessment, and the arterial blood gas sampling.\n8. Resting oxygen saturation (SpO2) \\>85% during 15 minutes without use of supplemental oxygen at the screening visits.\n9. Male or female\n10. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n\n 1. Male subjects: Males agreeing to use condoms during and until 30 days after last dose of trial treatment, or males having a female partner who is using adequate contraception as described below.\n 2. Female subjects: Females who have been post-menopausal for \\>1 year, or females of childbearing potential after a confirmed menstrual period using a highly efficient method of contraception (i.e. a method with \\<1% failure rate such as combined hormonal contraception, progesterone-only hormonal contraception, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence\\*), during and until 30 days after last dose of trial treatment. Females of childbearing potential must have a negative serum pregnancy test at the screening visits, and a negative urine pregnancy test at Baseline visit (Visit 3) and must not be lactating.\n11. Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n12. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other trial procedures specified in the protocol as judged by the Investigator.\n\nExclusion Criteria:\n\n1. Diagnosis of hereditary or secondary PAP, or a metabolic disorder of surfactant production.\n2. Whole lung lavage (WLL) performed within 3 months prior to baseline.\n3. Requirement for WLL at screening or baseline.\n4. GM-CSF treatment within 6 months prior to baseline.\n5. Treatment with rituximab within 6 months prior to baseline.\n6. Treatment with plasmapheresis within 6 weeks prior to baseline.\n7. Treatment with any investigational medicinal product within 5 half-lives or 3 months (whichever is longer) prior to baseline.\n8. Previously randomized in this trial.\n9. History of allergic reactions to GM-CSF or any of the excipients in the nebulizer solution.\n10. Inflammatory or autoimmune disease of a severity that necessitates significant (e.g. more than 10 mg/day systemic prednisolone) immunosuppression.\n11. Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product.\n12. History of, or present, myeloproliferative disease or leukemia.\n13. Apparent pre-existing concurrent pulmonary fibrosis.\n14. Acute or unstable cardiac or pulmonary disease that may be aggravated by exercise.\n15. Known active infection (viral, bacterial, fungal, or mycobacterial) that may affect the efficacy evaluation in the trial.\n16. Physical disability or other condition that precludes safe and adequate exercise testing.\n17. Any other serious medical condition which in the opinion of the Investigator would make the subject unsuitable for the trial.\n18. Pregnant, planning to become pregnant during the trial, or breastfeeding woman. For France only: including as further defined by French Health Code L-1121-5.\n19. For France only: Any subject considered to be "vulnerable" on account of, e.g., mental or physical disability, socio-economic situation, or subjects deprived of their liberty. For France only: including as further defined by French Health Code L1121-8-1.'}, 'identificationModule': {'nctId': 'NCT04544293', 'acronym': 'IMPALA-2', 'briefTitle': 'Clinical Trial of Inhaled Molgramostim Nebulizer Solution in Autoimmune Pulmonary Alveolar Proteinosis (aPAP)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Savara Inc.'}, 'officialTitle': 'A Randomized, Double-blind, Placebo-controlled Clinical Trial of Once-daily Inhaled Molgramostim Nebulizer Solution in Adult Subjects With Autoimmune Pulmonary Alveolar Proteinosis (aPAP)', 'orgStudyIdInfo': {'id': 'SAV006-05'}, 'secondaryIdInfos': [{'id': '2020-001263-85', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Molgramostim', 'description': 'Double-blind treatment with molgramostim nebulizer solution 300 µg once daily (Mol OD) for 48 weeks', 'interventionNames': ['Drug: Molgramostim']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Double-blind treatment with placebo (PBO) nebulizer solution once daily for 48 weeks', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Molgramostim Open-label Extension', 'description': 'Open-label treatment with molgramostim nebulizer solution 300 µg once daily (Mol OD) for 96 weeks', 'interventionNames': ['Drug: Molgramostim Open-label']}], 'interventions': [{'name': 'Molgramostim', 'type': 'DRUG', 'otherNames': ['Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF)'], 'description': 'Molgramostim 300 µg nebulizer solution', 'armGroupLabels': ['Molgramostim']}, {'name': 'Placebo', 'type': 'DRUG', 'otherNames': ['Placebo nebulizer'], 'description': 'Matching placebo nebulizer solution', 'armGroupLabels': ['Placebo']}, {'name': 'Molgramostim Open-label', 'type': 'DRUG', 'otherNames': ['Recombinant human granulocyte-macrophage colony stimulating factor (rhGM-CSF)'], 'description': 'Molgramostim 300 µg nebulizer solution', 'armGroupLabels': ['Molgramostim Open-label Extension']}]}, 'contactsLocationsModule': {'locations': [{'zip': '72205', 'city': 'Little Rock', 'state': 'Arkansas', 'country': 'United States', 'facility': 'University Of Arkansas For Medical Services', 'geoPoint': {'lat': 34.74648, 'lon': -92.28959}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'UCLA David Geffen School of Medicine', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '80206', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'National Jewish Health', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '06520', 'city': 'New Haven', 'state': 'Connecticut', 'country': 'United States', 'facility': 'Yale University', 'geoPoint': {'lat': 41.30815, 'lon': -72.92816}}, {'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida Health', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60153', 'city': 'Maywood', 'state': 'Illinois', 'country': 'United States', 'facility': 'Loyola University', 'geoPoint': {'lat': 41.8792, 'lon': -87.84312}}, {'zip': '21201', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'University of Maryland Medical Center', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University in St. Louis', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke University Medical Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '27610', 'city': 'Raleigh', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Wake Med Health & Hospital', 'geoPoint': {'lat': 35.7721, 'lon': -78.63861}}, {'zip': '45229-3039', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pennsylvania Perelman School of Medicine - Pulmonology', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'UT Southwestern Medical Center', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '2050', 'city': 'Camperdown', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Royal Prince Alfred Hospital', 'geoPoint': {'lat': -33.88965, 'lon': 151.17642}}, {'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': 'Westmead Hospital', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '1070', 'city': 'Brussels', 'state': 'Brussels Capital', 'country': 'Belgium', 'facility': 'Hôpital Erasme', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'zip': '3000', 'city': 'Leuven', 'state': 'Vlaams Brabant', 'country': 'Belgium', 'facility': 'UZ Leuven - 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