Ignite Creation Date:
2025-12-24 @ 2:29 PM
Ignite Modification Date:
2026-02-20 @ 10:45 PM
Study NCT ID:
NCT02107859
Status:
TERMINATED
Last Update Posted:
2020-03-25
First Post:
2014-04-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Study of Ataluren (PTC124) in Cystic Fibrosis
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Germany']}, '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': 'medinfo@ptcbio.com', 'phone': '1-866-562-4620', 'title': 'Patient Advocacy', 'organization': 'PTC Therapeutics, Inc.'}, 'certainAgreement': {'otherDetails': 'The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'Based on the results of study PTC124-GD-021-CF (NCT02139306) (did not achieve its primary or secondary endpoints), clinical development of ataluren in cystic fibrosis was discontinued and this ongoing open-label extension study was closed.'}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to end of study (Week 196)', 'description': 'As-treated population included all participants who received at least 1 dose of ataluren.', 'eventGroups': [{'id': 'EG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.', 'otherNumAtRisk': 61, 'deathsNumAtRisk': 61, 'otherNumAffected': 61, 'seriousNumAtRisk': 61, 'deathsNumAffected': 1, 'seriousNumAffected': 36}], 'otherEvents': [{'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 50}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Oral candidiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 22}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 8}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Haemorrhoids', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 9}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 12}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Musculoskeletal chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 4}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Drug hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 5}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 5}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}], 'seriousEvents': [{'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Cellulitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Clostridium difficile infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 27}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Dyspnoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 7}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Pneumothorax', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Distal ileal obstruction syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Distal intestinal obstruction syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Intestinal obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Blood creatine increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Forced expiratory volume decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Pulmonary function test decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Renal failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Renal failure acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Gallbladder pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Femur fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Colon cancer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}, {'term': 'Deep vein thrombosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 61, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 17.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'title': 'Any TEAEs', 'categories': [{'measurements': [{'value': '61', 'groupId': 'OG000'}]}]}, {'title': 'Mild AEs', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}, {'title': 'Moderate AEs', 'categories': [{'measurements': [{'value': '26', 'groupId': 'OG000'}]}]}, {'title': 'Severe AEs', 'categories': [{'measurements': [{'value': '30', 'groupId': 'OG000'}]}]}, {'title': 'Life-threatening AEs', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Fatal AEs', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'AEs unrelated to ataluren', 'categories': [{'measurements': [{'value': '35', 'groupId': 'OG000'}]}]}, {'title': 'AEs unlikely related to ataluren', 'categories': [{'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'AEs possible related to ataluren', 'categories': [{'measurements': [{'value': '13', 'groupId': 'OG000'}]}]}, {'title': 'AEs probable related to ataluren', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Serious TEAEs', 'categories': [{'measurements': [{'value': '36', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline (Day 1) up to end of study (Week 196)', 'description': 'AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'As-treated population included all participants who received at least 1 dose of ataluren.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Clinically Significant Laboratory Abnormalities', 'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline (Day 1) up to end of study (Week 196)', 'description': "Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'As-treated population included all participants who received at least 1 dose of ataluren.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '56.203', 'spread': '17.2964', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 192', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-1.214', 'spread': '3.6384', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 192', 'description': 'FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Percent of predicted FEV1 = (observed value)/(predicted value) \\* 100%. Change from baseline in percent predicted FEV1 at the end of treatment was reported.', 'unitOfMeasure': 'percentage of predicted FEV1', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "ITT population included all participants who had at least 1 post-baseline efficacy assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria', 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '68.3', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Week 192', 'description': "The modified Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms without the requirement for treatment with antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature greater than (\\>) 38 degrees celsius (°C); anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function.", 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who had at least 1 post-baseline efficacy assessment.'}, {'type': 'SECONDARY', 'title': "Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria", 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '68.3', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Week 192', 'description': "The expanded Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring any form of antibiotic treatment (inhaled, oral, or intravenous): change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature \\>38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function.", 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who had at least 1 post-baseline efficacy assessment.'}, {'type': 'SECONDARY', 'title': "Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria", 'denoms': [{'units': 'Participants', 'counts': [{'value': '60', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '58.3', 'groupId': 'OG000'}]}]}], 'paramType': 'NUMBER', 'timeFrame': 'Baseline up to Week 192', 'description': "The Classic Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring treatment with parenteral antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature \\>38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function.", 'unitOfMeasure': 'percentage of participants', 'reportingStatus': 'POSTED', 'populationDescription': 'ITT population included all participants who had at least 1 post-baseline efficacy assessment.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'title': 'Baseline: RR duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '828.17', 'spread': '132.15', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: RR duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-14.27', 'spread': '115.62', 'groupId': 'OG000'}]}]}, {'title': 'Baseline: PR duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '145.02', 'spread': '19.29', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: PR duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.69', 'spread': '12.25', 'groupId': 'OG000'}]}]}, {'title': 'Baseline: QRS duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '83.90', 'spread': '8.00', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: QRS duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.33', 'spread': '6.12', 'groupId': 'OG000'}]}]}, {'title': 'Baseline: QT duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '370.71', 'spread': '28.11', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: QT duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-4.38', 'spread': '25.63', 'groupId': 'OG000'}]}]}, {'title': 'Baseline: QTCB duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '408.92', 'spread': '22.05', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: QTCB duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.71', 'spread': '17.96', 'groupId': 'OG000'}]}]}, {'title': 'Baseline: QTCF duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '395.47', 'spread': '19.23', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: QTCF duration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.27', 'spread': '17.13', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 196', 'description': "ECG parameters included RR duration, PR duration, QRS duration, QT duration, QTCB (Bazett's correction formula) duration, QTCF (Fridericia's correction formula) duration.", 'unitOfMeasure': 'miiliseconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '74.31', 'spread': '12.06', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196', 'denoms': [{'units': 'Participants', 'counts': [{'value': '55', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '2.04', 'spread': '10.11', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 196', 'description': 'Heart rate was measured using 12-lead ECG.', 'unitOfMeasure': 'beats/minute', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable for specified categories."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Vital Signs at Final Visit (Week 196)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'title': 'Baseline: SBP', 'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '114.8', 'spread': '9.10', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: SBP', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.6', 'spread': '12.62', 'groupId': 'OG000'}]}]}, {'title': 'Baseline: DBP', 'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '71.2', 'spread': '8.93', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 196: DBP', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-0.3', 'spread': '9.93', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 196', 'description': 'Vital Signs included systolic blood pressure (SBP) and diastolic blood pressure (DBP).', 'unitOfMeasure': 'millimeters of mercury (mmHg)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "As-treated population included all participants who received at least 1 dose of ataluren. Here, 'Number analyzed' signifies participants evaluable for specified categories."}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '59', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '73.576', 'spread': '14.6552', 'groupId': 'OG000'}]}]}, {'title': 'Change at Week 192', 'denoms': [{'units': 'Participants', 'counts': [{'value': '7', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '-2.286', 'spread': '4.5722', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 192', 'description': 'FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position. Percent of predicted FVC = (observed value)/(predicted value) \\* 100%. Change from baseline in percent predicted FVC at the end of treatment was reported.', 'unitOfMeasure': 'percentage of predicted FVC', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "ITT population included all participants who had at least 1 post-baseline efficacy assessment. Here, 'Overall number of participants analyzed' signifies participants evaluable for this outcome measure. 'Number analyzed' signifies participants evaluable at specified timepoint."}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Change From Baseline in Percent Predicted Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) at the End of Treatment (Week 192), as Assessed by Spirometry', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'timeFrame': 'Baseline, Week 192', 'description': 'FEF25-75 is the forced expiratory flow between 25 and 75% of vital capacity.', 'reportingStatus': 'POSTED', 'populationDescription': 'Due to change in planned analysis FEV25-75 was not calculated and summarized.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '61'}]}, {'type': 'As-treated Population', 'comment': 'Received at .least 1 dose of study drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '61'}]}, {'type': 'Intent-to-treat (ITT) Population', 'comment': 'All participants who had at least 1 post-baseline efficacy assessment.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '60'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '61'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '14'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Study closure', 'reasons': [{'groupId': 'FG000', 'numSubjects': '41'}]}, {'type': 'Other than specified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}]}], 'recruitmentDetails': 'Participants with nonsense mutation cystic fibrosis (nmCF) who had completed the double-blind study PTC124-GD-009-CF (NCT00803205) were enrolled and treated in this open-label extension study.', 'preAssignmentDetails': 'On 2 March 2017, it was announced that the Phase 3 double-blind study PTC124-GD-021-CF (NCT02139306) did not achieve its primary or secondary endpoints. Based on these results, clinical development of ataluren in cystic fibrosis was discontinued and this ongoing open-label extension study was closed.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '61', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '27.5', 'spread': '10.73', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '34', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '27', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '60', 'groupId': 'BG000'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'categories': [{'title': 'White-White/Caucasian', 'measurements': [{'value': '61', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'As-treated population included all participants who received at least 1 dose of ataluren.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-07-14', 'size': 650830, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_000.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-03-11T12:11', 'hasProtocol': False}, {'date': '2016-02-08', 'size': 901581, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_001.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-03-11T12:12', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 61}}, 'statusModule': {'whyStopped': 'Based on the results of study PTC124-GD-021-CF (NCT02139306), clinical development of ataluren in cystic fibrosis was discontinued and this study was closed.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2014-05-23', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-03', 'dispFirstSubmitDate': '2019-01-24', 'completionDateStruct': {'date': '2017-06-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-03-11', 'studyFirstSubmitDate': '2014-04-04', 'dispFirstSubmitQcDate': '2019-01-24', 'resultsFirstSubmitDate': '2020-03-11', 'studyFirstSubmitQcDate': '2014-04-07', 'dispFirstPostDateStruct': {'date': '2019-01-28', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2020-03-25', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-03-11', 'studyFirstPostDateStruct': {'date': '2014-04-08', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-03-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-06-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at the End of Treatment (Week 192), as Assessed by Spirometry', 'timeFrame': 'Baseline, Week 192', 'description': 'FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position. Percent of predicted FVC = (observed value)/(predicted value) \\* 100%. Change from baseline in percent predicted FVC at the end of treatment was reported.'}, {'measure': 'Change From Baseline in Percent Predicted Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) at the End of Treatment (Week 192), as Assessed by Spirometry', 'timeFrame': 'Baseline, Week 192', 'description': 'FEF25-75 is the forced expiratory flow between 25 and 75% of vital capacity.'}], 'primaryOutcomes': [{'measure': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'timeFrame': 'Baseline (Day 1) up to end of study (Week 196)', 'description': 'AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.'}, {'measure': 'Number of Participants With Clinically Significant Laboratory Abnormalities', 'timeFrame': 'Baseline (Day 1) up to end of study (Week 196)', 'description': "Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement."}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry', 'timeFrame': 'Baseline, Week 192', 'description': 'FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Percent of predicted FEV1 = (observed value)/(predicted value) \\* 100%. Change from baseline in percent predicted FEV1 at the end of treatment was reported.'}, {'measure': 'Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria', 'timeFrame': 'Baseline up to Week 192', 'description': "The modified Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms without the requirement for treatment with antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature greater than (\\>) 38 degrees celsius (°C); anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function."}, {'measure': "Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria", 'timeFrame': 'Baseline up to Week 192', 'description': "The expanded Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring any form of antibiotic treatment (inhaled, oral, or intravenous): change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature \\>38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function."}, {'measure': "Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria", 'timeFrame': 'Baseline up to Week 192', 'description': "The Classic Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring treatment with parenteral antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature \\>38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function."}, {'measure': 'Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)', 'timeFrame': 'Baseline, Week 196', 'description': "ECG parameters included RR duration, PR duration, QRS duration, QT duration, QTCB (Bazett's correction formula) duration, QTCF (Fridericia's correction formula) duration."}, {'measure': 'Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG', 'timeFrame': 'Baseline, Week 196', 'description': 'Heart rate was measured using 12-lead ECG.'}, {'measure': 'Change From Baseline in Vital Signs at Final Visit (Week 196)', 'timeFrame': 'Baseline, Week 196', 'description': 'Vital Signs included systolic blood pressure (SBP) and diastolic blood pressure (DBP).'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Cystic fibrosis', 'Nonsense mutation', 'Premature stop codon', 'PTC124', 'Ataluren'], '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.'}], 'seeAlsoLinks': [{'url': 'http://www.ptcbio.com', 'label': 'PTC Therapeutics, Inc. website'}]}, 'descriptionModule': {'briefSummary': 'The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram \\[ECG\\] measurements, vital signs).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Ability to provide written informed consent (parental/guardian consent and participant assent if less than \\[\\<\\] 18 years of age).\n* Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).\n* Body weight greater than or equal to (≥) 16 kilograms (kg).\n* Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.\n* Confirmed laboratory values within the central laboratory ranges at screening.\n* In male and female participants 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, laboratory tests, and study restrictions.\n\nKey Exclusion Criteria:\n\n* Chronic use of systemic tobramycin within 4 weeks prior to screening.\n* Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.\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 CF or for CF-related conditions within 4 weeks prior to screening and randomization.\n* Known hypersensitivity to any of the ingredients or excipients of the study drug.\n* Exposure to another investigational drug within 4 weeks prior to screening.\n* Treatment with intravenous antibiotics within 3 weeks prior to screening.\n* History of solid organ or hematological transplantation.\n* Ongoing immunosuppressive therapy (other than corticosteroids).\n* Positive hepatitis B surface antigen, hepatitis C antibody test or human immunodeficiency virus (HIV) test.\n* Known portal hypertension.\n* Pregnancy or breast-feeding.'}, 'identificationModule': {'nctId': 'NCT02107859', 'briefTitle': 'Study of Ataluren (PTC124) in Cystic Fibrosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'PTC Therapeutics'}, 'officialTitle': 'An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)', 'orgStudyIdInfo': {'id': 'PTC124-GD-023-CF'}, 'secondaryIdInfos': [{'id': '2013-005449-35', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ataluren', 'description': 'Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks.', 'interventionNames': ['Drug: Ataluren']}], 'interventions': [{'name': 'Ataluren', 'type': 'DRUG', 'otherNames': ['PTC124'], 'description': 'Ataluren will be administered per dose and schedule specified in the arm.', 'armGroupLabels': ['Ataluren']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'University of Alabama-Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'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': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Denver Children's Hospital", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60614', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Children's Hospital Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Children's Hospital Boston", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10003', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Beth Israel Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '44106', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': "Rainbow Babies & Children's Hospital", 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'city': 'Brussels', 'country': 'Belgium', 'facility': 'Hôpital Universitaire des Enfants Reine Fabiola', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'city': 'Brussels', 'country': 'Belgium', 'facility': 'University Hospital Brussels', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'city': 'Leuven', 'country': 'Belgium', 'facility': 'University Hospital Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'city': 'Paris', 'country': 'France', 'facility': 'Hôpital Necker - Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '31059', 'city': 'Toulouse', 'country': 'France', 'facility': 'Hôpital des Enfants', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'zip': '91240', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Hadassah University Hospital - Mount Scopus', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'city': 'Roma', 'country': 'Italy', 'facility': 'Università La Sapienza', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'city': 'Verona', 'country': 'Italy', 'facility': 'Azienda Ospedaliera di Verona', 'geoPoint': {'lat': 45.43854, 'lon': 10.9938}}, {'city': 'Madrid', 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