Ignite Creation Date:
2025-12-26 @ 5:23 PM
Ignite Modification Date:
2026-03-03 @ 3:10 PM
Study NCT ID:
NCT02139306
Status:
COMPLETED
Last Update Posted:
2020-05-14
First Post:
2014-05-13
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C515878', 'term': 'ataluren'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'jmcintosh@ptcbio.com', 'phone': '908 912-9138', 'title': 'Joseph McIntosh', 'organization': 'PTC Therapeutics, Inc'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 Weeks)', 'description': 'All randomized participants who actually received any study treatment were analysed for AEs and SAEs.', 'eventGroups': [{'id': 'EG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.', 'otherNumAtRisk': 140, 'deathsNumAtRisk': 140, 'otherNumAffected': 123, 'seriousNumAtRisk': 140, 'deathsNumAffected': 0, 'seriousNumAffected': 40}, {'id': 'EG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.', 'otherNumAtRisk': 139, 'deathsNumAtRisk': 139, 'otherNumAffected': 120, 'seriousNumAtRisk': 139, 'deathsNumAffected': 0, 'seriousNumAffected': 46}], 'otherEvents': [{'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 15, 'numAffected': 13}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 20, 'numAffected': 12}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 14, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 12, 'numAffected': 10}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 15, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 6, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 8, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 7, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 17, 'numAffected': 9}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 12, 'numAffected': 10}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 142, 'numAffected': 75}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 145, 'numAffected': 78}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 43, 'numAffected': 27}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 25, 'numAffected': 21}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 25, 'numAffected': 21}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 26, 'numAffected': 21}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 20, 'numAffected': 16}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 11, 'numAffected': 11}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 13, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 10, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 12, 'numAffected': 10}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 8, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pseudomonas infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 10, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Staphylococcal infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 9, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 7, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 7, 'numAffected': 3}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 23, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 18, 'numAffected': 16}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 36, 'numAffected': 25}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 31, 'numAffected': 25}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 17, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 22, 'numAffected': 10}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}], 'seriousEvents': [{'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 36, 'numAffected': 25}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 50, 'numAffected': 34}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pseudomonas infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 2, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Aspergillus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Bronchopneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Bronchopulmonary aspergillosis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Device related sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Peritonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pneumonia influenzal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 2, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Mycobacterium abscessus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pneumonia staphylococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Respiratory tract infection fungal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Respiratory tract infection viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 2, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 4, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 2, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Nasal polyps', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Tachypnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Bronchopneumopathy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pneumothorax spontaneous', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Distal intestinal obstruction syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Small intestinal obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Renal failure acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pulmonary function test decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 3, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Forced expiratory volume decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Diabetes mellitus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Syncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Red man syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Skin and subcutaneous tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Pseudocholinesterase deficiency', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Thrombosis in device', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Costochondritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}, {'term': 'Suicidal ideation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 140, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 139, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.0)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '138', 'groupId': 'OG000'}, {'value': '136', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'categories': [{'measurements': [{'value': '-1.396', 'groupId': 'OG000', 'lowerLimit': '-2.7735', 'upperLimit': '-0.0180'}, {'value': '-1.992', 'groupId': 'OG001', 'lowerLimit': '-3.3271', 'upperLimit': '-0.6576'}]}]}], 'analyses': [{'pValue': '0.5336', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.597', 'ciLowerLimit': '-1.2881', 'ciUpperLimit': '2.4813', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.9570', 'statisticalMethod': 'Mixed-model, repeated-measures', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'From Baseline to Week 48', 'description': 'The FEV1 is the volume of air forcibly exhaled in one second and is measured using forced expiratory air spirometry. Change in ppFEV1 at Week 48 was defined as the average between the change from baseline at Week 40 and that at Week 48. Baseline for ppFEV1 was defined as an average of ppFEV1 at Screening (Weeks -4 to -1) and Baseline (Day 1) visits.', 'unitOfMeasure': 'Percentage of predicted FEV1', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The intent-to-treat (ITT) population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.'}, {'type': 'SECONDARY', 'title': '48-week Rate of Pulmonary Exacerbations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '138', 'groupId': 'OG000'}, {'value': '136', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.950', 'spread': '1.4038', 'groupId': 'OG000'}, {'value': '1.127', 'spread': '2.5241', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.4008', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Rate ratio', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.8567', 'ciLowerLimit': '0.5973', 'ciUpperLimit': '1.2288', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.1577', 'statisticalMethod': 'Negative binomial regression', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Week 48', 'description': "Pulmonary exacerbations were assessed using expanded Fuchs criteria. The expanded Fuchs exacerbation is defined as the presence of at least 4 of 12 Fuchs' signs and symptoms requiring treatment with any form of antibiotic treatment (inhaled, oral, or intravenous). Fuchs' signs and symptoms included increased cough; change in sputum volume, color, or consistency; new or increased hemoptysis; increased dyspnea during moderate or mild exertion, or at rest; sinus pain or tenderness; change in sinus discharge; malaise, fatigue, or lethargy; anorexia or weight loss; temperature above 38 degrees Celsius; change in findings on chest examination; relative 10% decrease in ppFEV1, and chest radiography results consistent with pulmonary infection. The 48-week rate was calculated as: 48-week rate = total number of events /treatment duration by week\\*48.", 'unitOfMeasure': 'number of exacerbations per 48 weeks', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '138', 'groupId': 'OG000'}, {'value': '136', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.760', 'groupId': 'OG000', 'lowerLimit': '-3.4566', 'upperLimit': '1.9364'}, {'value': '-1.032', 'groupId': 'OG001', 'lowerLimit': '-3.7368', 'upperLimit': '1.6728'}]}]}], 'analyses': [{'pValue': '0.8881', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.272', 'ciLowerLimit': '-3.5292', 'ciUpperLimit': '4.0731', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '1.9300', 'statisticalMethod': 'Mixed-model, repeated measures', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and Week 48', 'description': 'The teen/adult CFQ-R was used for this study. It was developed specifically for participants with cystic fibrosis. It is a disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being, and symptoms. The respiratory domain assessed respiratory symptoms like coughing, congestion, wheezing etc. Scaling of each item is done via 4-point Likert scales. Scores for each item are summed up to generate a domain score. Scores ranges from 0 to 100, with higher scores indicating better health and lower scores indicating worse health.', 'unitOfMeasure': 'units on a scale', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Body Mass Index (BMI) at Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '138', 'groupId': 'OG000'}, {'value': '136', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.296', 'groupId': 'OG000', 'lowerLimit': '0.1126', 'upperLimit': '0.4789'}, {'value': '0.361', 'groupId': 'OG001', 'lowerLimit': '0.1759', 'upperLimit': '0.5455'}]}]}], 'analyses': [{'pValue': '0.6208', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Net)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-0.065', 'ciLowerLimit': '-0.3233', 'ciUpperLimit': '0.1934', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.1312', 'statisticalMethod': 'Mixed-model, repeated measures', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline (Day 1) and Week 48', 'description': 'Malnutrition is common in participants with cystic fibrosis. The BMI is an important clinical measure of nutritional status.', 'unitOfMeasure': 'kilogram per meter square (kg/m^2)', 'dispersionType': '95% Confidence Interval', 'reportingStatus': 'POSTED', 'populationDescription': 'The ITT population included all randomized participants who had FEV1 data available at Baseline and at least one post-baseline visit.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}, {'value': '139', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'title': 'TEAEs', 'categories': [{'measurements': [{'value': '133', 'groupId': 'OG000'}, {'value': '135', 'groupId': 'OG001'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '40', 'groupId': 'OG000'}, {'value': '46', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'An adverse event (AE) is any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study treatment, whether or not considered related to the study treatment. A TEAE is defined as an AE that occurs or worsens in the period extending from first dose of study drug to 4 weeks after last dose of study drug. An SAE is an untoward medical occurrence or effect associated with the use of a study drug at any dose, regardless of whether it is considered to be related to the study drug, which results in one of the following: death; inpatient hospitalization or prolongation of existing hospitalization; life threatening adverse event; persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions; any other medically important event; or a pregnancy resulting in spontaneous abortion, stillbirth, neonatal death, or congenital anomaly.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The as-treated population included all randomized participants who actually received any study treatment.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With TEAEs by Severity and Relationship to Study Drugs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}, {'value': '139', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'title': 'Severity: Grade 1', 'categories': [{'measurements': [{'value': '26', 'groupId': 'OG000'}, {'value': '18', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 2', 'categories': [{'measurements': [{'value': '88', 'groupId': 'OG000'}, {'value': '88', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 3', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}, {'value': '29', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 4', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 5', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Unrelated', 'categories': [{'measurements': [{'value': '74', 'groupId': 'OG000'}, {'value': '72', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Unlikely related', 'categories': [{'measurements': [{'value': '37', 'groupId': 'OG000'}, {'value': '34', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Possible related', 'categories': [{'measurements': [{'value': '21', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Probable related', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'The relationship of TEAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of TEAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The as-treated population included all randomized participants who actually received any study treatment.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With SAEs by Severity and Relationship to Study Drugs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}, {'value': '139', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'title': 'Severity: Grade 1', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 2', 'categories': [{'measurements': [{'value': '23', 'groupId': 'OG000'}, {'value': '20', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 3', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}, {'value': '25', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 4', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Severity: Grade 5', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Unrelated', 'categories': [{'measurements': [{'value': '26', 'groupId': 'OG000'}, {'value': '31', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Unlikely related', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Possible related', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'Relationship to study drug: Probable related', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'The relationship of SAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of SAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The as-treated population included all randomized participants who actually received any study treatment.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Abnormal Vital Signs Reported as TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}, {'value': '139', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'Vital signs included systolic and diastolic blood pressure, pulse rate, pulse oximetry, and body temperature. Participants with abnormal vital signs who required clinical intervention or further investigation (beyond ordering a repeat \\[confirmatory\\] test) unless they are associated with an already reported clinical event are reported.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The as-treated population included all randomized participants who actually received any study treatment.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}, {'value': '139', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'categories': [{'measurements': [{'value': '32', 'groupId': 'OG000'}, {'value': '30', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'Clinical laboratory tests included haematology, biochemistry, and urinalysis. Participants with abnormal laboratory parameters who required clinical intervention or further investigation (beyond ordering a repeat \\[confirmatory\\] test) unless they are associated with an already reported clinical event are reported.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The as-treated population included all randomized participants who actually received any study treatment.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Abnormal Electrocardiogram Reported as TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'OG000'}, {'value': '139', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'OG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'Participants with abnormal electrocardiogram who required clinical intervention or further investigation (beyond ordering a repeat \\[confirmatory\\] test) unless they are associated with an already reported clinical event are reported.', 'unitOfMeasure': 'participants', 'reportingStatus': 'POSTED', 'populationDescription': 'The as-treated population included all randomized participants who actually received any study treatment.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'FG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '140'}, {'groupId': 'FG001', 'numSubjects': '139'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '127'}, {'groupId': 'FG001', 'numSubjects': '125'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '13'}, {'groupId': 'FG001', 'numSubjects': '14'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '6'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '4'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Other Unspecified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '3'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'This study was conducted from 15 August 2014 to 02 November 2016.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '140', 'groupId': 'BG000'}, {'value': '139', 'groupId': 'BG001'}, {'value': '279', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Ataluren', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation'}, {'id': 'BG001', 'title': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '22.0', 'spread': '11.0', 'groupId': 'BG000'}, {'value': '22.0', 'spread': '10.44', 'groupId': 'BG001'}, {'value': '22.0', 'spread': '10.70', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '59', 'groupId': 'BG000'}, {'value': '74', 'groupId': 'BG001'}, {'value': '133', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '81', 'groupId': 'BG000'}, {'value': '65', 'groupId': 'BG001'}, {'value': '146', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'The as-treated population included all randomized participants who actually received any study treatment.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 279}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2014-08', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-05', 'completionDateStruct': {'date': '2016-11', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-05-04', 'studyFirstSubmitDate': '2014-05-13', 'resultsFirstSubmitDate': '2017-05-30', 'studyFirstSubmitQcDate': '2014-05-14', 'lastUpdatePostDateStruct': {'date': '2020-05-14', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-05-04', 'studyFirstPostDateStruct': {'date': '2014-05-15', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-05-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2016-11', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48', 'timeFrame': 'From Baseline to Week 48', 'description': 'The FEV1 is the volume of air forcibly exhaled in one second and is measured using forced expiratory air spirometry. Change in ppFEV1 at Week 48 was defined as the average between the change from baseline at Week 40 and that at Week 48. Baseline for ppFEV1 was defined as an average of ppFEV1 at Screening (Weeks -4 to -1) and Baseline (Day 1) visits.'}], 'secondaryOutcomes': [{'measure': '48-week Rate of Pulmonary Exacerbations', 'timeFrame': 'Week 48', 'description': "Pulmonary exacerbations were assessed using expanded Fuchs criteria. The expanded Fuchs exacerbation is defined as the presence of at least 4 of 12 Fuchs' signs and symptoms requiring treatment with any form of antibiotic treatment (inhaled, oral, or intravenous). Fuchs' signs and symptoms included increased cough; change in sputum volume, color, or consistency; new or increased hemoptysis; increased dyspnea during moderate or mild exertion, or at rest; sinus pain or tenderness; change in sinus discharge; malaise, fatigue, or lethargy; anorexia or weight loss; temperature above 38 degrees Celsius; change in findings on chest examination; relative 10% decrease in ppFEV1, and chest radiography results consistent with pulmonary infection. The 48-week rate was calculated as: 48-week rate = total number of events /treatment duration by week\\*48."}, {'measure': 'Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48', 'timeFrame': 'Baseline (Day 1) and Week 48', 'description': 'The teen/adult CFQ-R was used for this study. It was developed specifically for participants with cystic fibrosis. It is a disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being, and symptoms. The respiratory domain assessed respiratory symptoms like coughing, congestion, wheezing etc. Scaling of each item is done via 4-point Likert scales. Scores for each item are summed up to generate a domain score. Scores ranges from 0 to 100, with higher scores indicating better health and lower scores indicating worse health.'}, {'measure': 'Change From Baseline in Body Mass Index (BMI) at Week 48', 'timeFrame': 'Baseline (Day 1) and Week 48', 'description': 'Malnutrition is common in participants with cystic fibrosis. The BMI is an important clinical measure of nutritional status.'}, {'measure': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'An adverse event (AE) is any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study treatment, whether or not considered related to the study treatment. A TEAE is defined as an AE that occurs or worsens in the period extending from first dose of study drug to 4 weeks after last dose of study drug. An SAE is an untoward medical occurrence or effect associated with the use of a study drug at any dose, regardless of whether it is considered to be related to the study drug, which results in one of the following: death; inpatient hospitalization or prolongation of existing hospitalization; life threatening adverse event; persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions; any other medically important event; or a pregnancy resulting in spontaneous abortion, stillbirth, neonatal death, or congenital anomaly.'}, {'measure': 'Number of Participants With TEAEs by Severity and Relationship to Study Drugs', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'The relationship of TEAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of TEAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).'}, {'measure': 'Number of Participants With SAEs by Severity and Relationship to Study Drugs', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'The relationship of SAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of SAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).'}, {'measure': 'Number of Participants With Abnormal Vital Signs Reported as TEAEs', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'Vital signs included systolic and diastolic blood pressure, pulse rate, pulse oximetry, and body temperature. Participants with abnormal vital signs who required clinical intervention or further investigation (beyond ordering a repeat \\[confirmatory\\] test) unless they are associated with an already reported clinical event are reported.'}, {'measure': 'Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'Clinical laboratory tests included haematology, biochemistry, and urinalysis. Participants with abnormal laboratory parameters who required clinical intervention or further investigation (beyond ordering a repeat \\[confirmatory\\] test) unless they are associated with an already reported clinical event are reported.'}, {'measure': 'Number of Participants With Abnormal Electrocardiogram Reported as TEAEs', 'timeFrame': 'From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)', 'description': 'Participants with abnormal electrocardiogram who required clinical intervention or further investigation (beyond ordering a repeat \\[confirmatory\\] test) unless they are associated with an already reported clinical event are reported.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'references': [{'pmid': '36866921', 'type': 'DERIVED', 'citation': 'Aslam AA, Sinha IP, Southern KW. Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2023 Mar 3;3(3):CD012040. doi: 10.1002/14651858.CD012040.pub3.'}, {'pmid': '32682670', 'type': 'DERIVED', 'citation': 'VanDevanter DR, Hamblett NM, Simon N, McIntosh J, Konstan MW. Evaluating assumptions of definition-based pulmonary exacerbation endpoints in cystic fibrosis clinical trials. J Cyst Fibros. 2021 Jan;20(1):39-45. doi: 10.1016/j.jcf.2020.07.008. Epub 2020 Jul 15.'}, {'pmid': '31983658', 'type': 'DERIVED', 'citation': 'Konstan MW, VanDevanter DR, Rowe SM, Wilschanski M, Kerem E, Sermet-Gaudelus I, DiMango E, Melotti P, McIntosh J, De Boeck K; ACT CF Study Group. Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF). J Cyst Fibros. 2020 Jul;19(4):595-601. doi: 10.1016/j.jcf.2020.01.007. Epub 2020 Jan 23.'}], 'seeAlsoLinks': [{'url': 'http://www.ptcbio.com', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'This is a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.', 'detailedDescription': 'This study is to enroll 208 subjects (184 fully evaluable) with nonsense-mutation-mediated CF who are at least 6 years of age and have an forced expiratory volume in 1 second (FEV1) \\>= 40% and \\<= 90% of predicted. Subjects will be stratified based on age, inhaled antibiotic use, and baseline FEV1, and will be randomized in a 1:1 ratio to receive oral ataluren administered 3 times per day (TID) at respective morning, midday, and evening doses of 10-, 10-, and 20-mg/kg or placebo. Based on the results of a previously conducted study, patients treated with chronic inhaled aminoglycosides (including TOBI) will not be eligible for participation. Spirometry measurement at the screening visit will establish patient eligibility for inclusion based on lung function. FEV1 stability will be assessed during the approximately 4-week screening period, at the conclusion of which patients will be required to demonstrate a relative change in %-predicted FEV1 of less than 15% when compared to the screening value. Assessments will be performed every 8 weeks, depending upon the outcome measure.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial\n* Age \\>=6 years.\n* Body weight \\>=16 kg.\n* Sweat chloride \\>60 milliequivalent per liter (mEq/L)\n* Documentation of the presence of a nonsense mutation in at least 1 allele of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, as determined by genotyping performed at a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization\n* Verification that a blood sample has been drawn for sequencing of the CFTR gene\n* Ability to perform a valid, reproducible spirometry test using the study-specific spirometer with demonstration of an FEV1 \\>=40% and \\<=90% of predicted\n* Demonstration at Visit 2 of a valid %-predicted FEV1 within 15% of the Screening % predicted FEV1 value\n* Resting oxygen saturation (as measured by pulse oximetry) \\>=92% on room air.\n* Confirmed screening laboratory values within pre-specified ranges\n* In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period\n* Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions\n\nExclusion Criteria:\n\n* Known hypersensitivity to any of the ingredients or excipients of the study drug\n* Previous participation in the Phase 3 trial of ataluren (PTC124-GD-009-CF).\n* Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for Cystic Fibrosis (CF) or for CF-related conditions within 4 weeks prior to screening\n* Chronic use of inhaled aminoglycosides (eg, tobramycin) or use of inhaled aminoglycosides within 4 weeks prior to screening.\n* Exposure to another investigational drug within 4 weeks prior to screening\n* Ongoing participation in any other therapeutic clinical trial\n* Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening\n* Treatment with intravenous antibiotics within 3 weeks prior to screening\n* Ongoing immunosuppressive therapy (other than corticosteroids)\n* Ongoing warfarin, phenytoin, or tolbutamide therapy\n* History of solid organ or hematological transplantation\n* Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to screening\n* Known portal hypertension\n* Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test\n* Pregnancy or breast-feeding\n* Current smoker or a smoking history of \\>=10 pack-years (number of cigarette packs/day x number of years smoked).\n* Prior or ongoing medical condition (eg, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results"}, 'identificationModule': {'nctId': 'NCT02139306', 'briefTitle': 'Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)', 'organization': {'class': 'INDUSTRY', 'fullName': 'PTC Therapeutics'}, 'officialTitle': 'A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis', 'orgStudyIdInfo': {'id': 'PTC124-GD-021-CF'}, 'secondaryIdInfos': [{'id': '2013-004581-34', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ataluren (PTC124®)', 'description': 'Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.', 'interventionNames': ['Drug: Ataluren (PTC124®)']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': 'Ataluren (PTC124®)', 'type': 'DRUG', 'description': 'Oral Ataluren TID', 'armGroupLabels': ['Ataluren (PTC124®)']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Oral Placebo TID', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '36608', 'city': 'Mobile', 'state': 'Alabama', 'country': 'United States', 'facility': 'Pulmonary Associates of Mobile PC', 'geoPoint': {'lat': 30.69436, 'lon': -88.04305}}, {'zip': '90806', 'city': 'Long Beach', 'state': 'California', 'country': 'United States', 'facility': "Miller Children's Hospital Long Beach", 'geoPoint': {'lat': 33.76696, 'lon': -118.18923}}, {'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital Los Angeles", 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94609', 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