Ignite Creation Date:
2025-12-24 @ 7:42 PM
Ignite Modification Date:
2025-12-29 @ 2:36 PM
Study NCT ID:
NCT02456103
Status:
TERMINATED
Last Update Posted:
2020-04-27
First Post:
2015-05-26
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
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': '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': 'This study was terminated early because the CF data from the double-blind CF Study PTC124-GD-021-CF (NCT02139306) did not meet endpoints.'}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to Week 100', 'description': 'Adverse events collected from participants who received at least 1 dose of ataluren (As-Treated Population).', 'eventGroups': [{'id': 'EG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.', 'otherNumAtRisk': 245, 'deathsNumAtRisk': 245, 'otherNumAffected': 191, 'seriousNumAtRisk': 245, 'deathsNumAffected': 1, 'seriousNumAffected': 89}], 'otherEvents': [{'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 136}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 13}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 25}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 21}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 30}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 32}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 14}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Forced expiratory volume decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 16}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 16}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 14}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 13}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}], 'seriousEvents': [{'term': 'Bronchopulmonary aspergillosis allergic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Infective pulmonary exacerbation of cystic fibrosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 63}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Mycobacterium abscessus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Peritonitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Pseudomonas infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Respiratory syncytial virus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Viral pericarditis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Abdominal pain lower', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Distal intestinal obstruction syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Duodenal ulcer', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Enteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Intestinal obstruction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Pancreatitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Acute respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Asthma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Dyspnoea exertional', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Haemoptysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Forced expiratory volume decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Protein urine present', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Pseudomonas test positive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Pulmonary function test decreased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Renal colic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 2}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Renal failure acute', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Cystic fibrosis lung', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Squamous cell carcinoma of the cervix', 'notes': 'This is a sex-specific adverse event that only affects female participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 113, 'numAffected': 1}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Depressed level of consciousness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Pregnancy', 'notes': 'This is a sex-specific adverse event that only affects female participants.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 113, 'numAffected': 1}], 'organSystem': 'Pregnancy, puerperium and perinatal conditions', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 245, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': '17.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '245', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'classes': [{'title': 'At least 1 TEAE', 'categories': [{'measurements': [{'value': '222', 'groupId': 'OG000'}]}]}, {'title': 'Mild TEAE', 'categories': [{'measurements': [{'value': '28', 'groupId': 'OG000'}]}]}, {'title': 'Moderate TEAE', 'categories': [{'measurements': [{'value': '147', 'groupId': 'OG000'}]}]}, {'title': 'Severe TEAE', 'categories': [{'measurements': [{'value': '46', 'groupId': 'OG000'}]}]}, {'title': 'Life-Threatening TEAE', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Fatal TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'Serious TEAE', 'categories': [{'measurements': [{'value': '89', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Unrelated to Study Drug', 'categories': [{'measurements': [{'value': '140', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Unlikely Related to Study Drug', 'categories': [{'measurements': [{'value': '55', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Possibly Related to Study Drug', 'categories': [{'measurements': [{'value': '23', 'groupId': 'OG000'}]}]}, {'title': 'TEAE Probably Related to Study Drug', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 100', 'description': "TEAE: any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. Serious adverse event (SAE): an adverse event (AE) resulting in any of following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn't reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who received at least 1 dose of ataluren (As-Treated Population).'}, {'type': 'PRIMARY', 'title': 'Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter', 'denoms': [{'units': 'Participants', 'counts': [{'value': '245', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 100', 'description': 'Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: white blood cell count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who received at least 1 dose of ataluren (As-Treated Population).'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '233', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '233', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '60.219', 'spread': '17.6163', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '205', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.015', 'spread': '6.7180', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Pulmonary function of percent-predicted FEV1 was measured using a spirometer. FEV1 is the volume of air that can forcibly be blown out in 1 second. Each percent-predicted FEV1 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEV1 was calculated as follows: (percent-predicted FEV1 - Baseline percent-predicted FEV1/Baseline percent-predicted FEV1)\\*100.', 'unitOfMeasure': 'percentage of predicted FEV1', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEV1 data.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '233', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '233', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '75.249', 'spread': '15.8508', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '205', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.166', 'spread': '6.5276', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Pulmonary function of FVC was measured using a spirometer. FVC is the volume of air that can forcibly be blown out. Each percent-predicted FVC was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FVC was calculated as follows: (percent-predicted FVC - Baseline percent-predicted FVC/Baseline percent-predicted FVC)\\*100.', 'unitOfMeasure': 'percentage of predicted FVC', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FVC data.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '233', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'classes': [{'title': 'Baseline', 'denoms': [{'units': 'Participants', 'counts': [{'value': '233', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '38.099', 'spread': '22.0980', 'groupId': 'OG000'}]}]}, {'title': 'Change from Baseline at Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '205', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '0.698', 'spread': '13.1241', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 24', 'description': 'Pulmonary function of FEF25-75 was measured using a spirometer. FEF25-75 is the forced expiratory flow between 25% and 75% of vital capacity. Each percent-predicted FEF25-75 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEF25-75 was calculated as follows: (percent-predicted FEF25-75 - Baseline percent-predicted FEF25-75/Baseline percent-predicted FEF25-75)\\*100.', 'unitOfMeasure': 'percentage of FEF25-75', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population) and had evaluable FEF25-75 data.'}, {'type': 'SECONDARY', 'title': "Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks", 'denoms': [{'units': 'Participants', 'counts': [{'value': '244', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.051', 'spread': '2.1654', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline up to Week 48', 'description': "A modified Fuchs' exacerbation was defined as an event requiring treatment with or without intravenous antibiotics for any 4 of the following 12 symptoms: 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. The 48-week rate = (the total number of events/ treatment duration by week)\\*48.", 'unitOfMeasure': 'exacerbations', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Participants who received at least 1 dose of ataluren and have at least 1 postbaseline efficacy assessment (ITT Population).'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '246'}]}, {'type': 'As-Treated Population', 'comment': 'Participants who received at least 1 dose of ataluren.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '245'}]}, {'type': 'Intent-to-treat (ITT) Population', 'comment': 'Participants in the As-Treated Population with at least 1 postbaseline efficacy assessment.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '244'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '246'}]}], 'dropWithdraws': [{'type': 'Study Closure', 'reasons': [{'groupId': 'FG000', 'numSubjects': '207'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Required prohibited medications', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Protocol Violation', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}]}, {'type': 'Abnormal Laboratory Value', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '24'}]}]}], 'preAssignmentDetails': 'All eligible participants, including those who received placebo in the double-blind study (PTC124-GD-021-CF; NCT02139306), were enrolled to this open-label extension study. To avoid interruption in treatment, when possible, Screening/Baseline for this extension study was to occur on the same day as the End-of-Study visit for the double-blind study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '245', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ataluren', 'description': 'Participants were administered ataluren orally at a dose of 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '23.1', 'spread': '10.88', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '113', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '132', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Asian', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}, {'title': 'White', 'categories': [{'measurements': [{'value': '235', 'groupId': 'BG000'}]}]}, {'title': 'White-Arabic/North African Heritage', 'categories': [{'measurements': [{'value': '1', 'groupId': 'BG000'}]}]}, {'title': 'Non-White', 'categories': [{'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}, {'title': 'Hispanic or Latino', 'categories': [{'measurements': [{'value': '16', 'groupId': 'BG000'}]}]}, {'title': 'Not Hispanic or Latino', 'categories': [{'measurements': [{'value': '229', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'Participants who received at least 1 dose of ataluren (As-Treated Population).'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2015-03-27', 'size': 893297, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-04-01T16:58', 'hasProtocol': True}, {'date': '2017-07-14', 'size': 615999, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-04-01T16:58', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 246}}, 'statusModule': {'whyStopped': 'Cystic Fibrosis (CF) data from the double-blind CF Study PTC124-GD-021-CF did not meet endpoints.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2015-08-31', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-04', 'dispFirstSubmitDate': '2019-01-24', 'completionDateStruct': {'date': '2017-06-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-04-14', 'studyFirstSubmitDate': '2015-05-26', 'dispFirstSubmitQcDate': '2019-01-24', 'resultsFirstSubmitDate': '2020-04-01', 'studyFirstSubmitQcDate': '2015-05-26', 'dispFirstPostDateStruct': {'date': '2019-01-28', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2020-04-27', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-04-01', 'studyFirstPostDateStruct': {'date': '2015-05-28', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-04-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-06-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs)', 'timeFrame': 'Baseline up to Week 100', 'description': "TEAE: any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. Serious adverse event (SAE): an adverse event (AE) resulting in any of following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn't reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section."}, {'measure': 'Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter', 'timeFrame': 'Baseline up to Week 100', 'description': 'Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: white blood cell count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Pulmonary function of percent-predicted FEV1 was measured using a spirometer. FEV1 is the volume of air that can forcibly be blown out in 1 second. Each percent-predicted FEV1 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEV1 was calculated as follows: (percent-predicted FEV1 - Baseline percent-predicted FEV1/Baseline percent-predicted FEV1)\\*100.'}, {'measure': 'Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Pulmonary function of FVC was measured using a spirometer. FVC is the volume of air that can forcibly be blown out. Each percent-predicted FVC was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FVC was calculated as follows: (percent-predicted FVC - Baseline percent-predicted FVC/Baseline percent-predicted FVC)\\*100.'}, {'measure': 'Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24', 'timeFrame': 'Baseline, Week 24', 'description': 'Pulmonary function of FEF25-75 was measured using a spirometer. FEF25-75 is the forced expiratory flow between 25% and 75% of vital capacity. Each percent-predicted FEF25-75 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEF25-75 was calculated as follows: (percent-predicted FEF25-75 - Baseline percent-predicted FEF25-75/Baseline percent-predicted FEF25-75)\\*100.'}, {'measure': "Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks", 'timeFrame': 'Baseline up to Week 48', 'description': "A modified Fuchs' exacerbation was defined as an event requiring treatment with or without intravenous antibiotics for any 4 of the following 12 symptoms: 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. The 48-week rate = (the total number of events/ treatment duration by week)\\*48."}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': 'This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.', 'detailedDescription': 'The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)\n* Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.\n\nExclusion Criteria:\n\n* Known hypersensitivity to any of the ingredients or excipients of the study drug.\n* Ongoing participation in any other therapeutic clinical trial."}, 'identificationModule': {'nctId': 'NCT02456103', 'briefTitle': 'Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'PTC Therapeutics'}, 'officialTitle': 'Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis', 'orgStudyIdInfo': {'id': 'PTC124-GD-021e-CF'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ataluren', 'description': 'Participants will be administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.', 'interventionNames': ['Drug: Ataluren']}], 'interventions': [{'name': 'Ataluren', 'type': 'DRUG', 'otherNames': ['PTC124', 'Translarna'], 'description': 'Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.', 'armGroupLabels': ['Ataluren']}]}, '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': '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', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital and Research Center at Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': "Stanford University-Children's Hospital", 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '32207', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': "Nemours Children's Clinic", 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '33155', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': "Miami Children's Hospital", 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '33701', 'city': 'St. Petersburg', 'state': 'Florida', 'country': 'United States', 'facility': "All Children's Hospital", 'geoPoint': {'lat': 27.77086, 'lon': -82.67927}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann and Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Children's Hospital Boston", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '07960', 'city': 'Morristown', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Morristown Medical Center', 'geoPoint': {'lat': 40.79677, 'lon': -74.48154}}, {'zip': '10011', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Beth Israel Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10016', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'New York University Langone Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '45221', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'University of Cincinnati', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '17033', 'city': 'Hershey', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Penn State Milton S. Hershey Medical Center', 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '19129', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Drexel University College of Medicine', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '77094', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '75708', 'city': 'Tyler', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Health Science Center', 'geoPoint': {'lat': 32.35126, 'lon': -95.30106}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Childrens Hospital of Wisconsin', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '1330', 'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hospital de Ninos Ricardo Gutierrez', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'city': 'Buenos Aires', 'country': 'Argentina', 'facility': 'Hospital Universitario Austral', 'geoPoint': {'lat': -34.61315, 'lon': -58.37723}}, {'zip': '5000', 'city': 'Adelaide', 'country': 'Australia', 'facility': 'Royal Adelaide Hospital', 'geoPoint': {'lat': -34.92866, 'lon': 138.59863}}, {'zip': '4032', 'city': 'Chermside', 'country': 'Australia', 'facility': 'Prince Charles Hospital', 'geoPoint': {'lat': -27.38472, 'lon': 153.03062}}, {'zip': '6840', 'city': 'Perth', 'country': 'Australia', 'facility': 'Princess Margaret Hospital', 'geoPoint': {'lat': -31.95224, 'lon': 115.8614}}, {'zip': '1090', 'city': 'Brussels', 'country': 'Belgium', 'facility': 'University Hospital Brussels', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'zip': '15', 'city': 'Brussels', 'country': 'Belgium', 'facility': 'Hopital Universitaire des Enfants Reine Fabiola', 'geoPoint': {'lat': 50.85045, 'lon': 4.34878}}, {'zip': '3000', 'city': 'Leuven', 'country': 'Belgium', 'facility': 'University Hospital Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'city': 'Porto Alegre', 'country': 'Brazil', 'facility': 'Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul', 'geoPoint': {'lat': -30.03283, 'lon': -51.23019}}, {'city': 'Plovdiv', 'country': 'Bulgaria', 'facility': 'University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD', 'geoPoint': {'lat': 42.15387, 'lon': 24.75001}}, {'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'University Multiprofile Hospital for Active Treatment Aleksandrovska EAD', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'zip': 'H2W 1R7', 'city': 'Montreal', 'country': 'Canada', 'facility': 'Clinical Research Institute of Montreal', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'country': 'Canada', 'facility': 'University of Toronto Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'V6H 3V4', 'city': 'Vancouver', 'country': 'Canada', 'facility': "British Columbia Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': '34295', 'city': 'Montpellier', 'country': 'France', 'facility': 'Hoptial Arnaud de Villeneuve', 'geoPoint': {'lat': 43.61093, 'lon': 3.87635}}, {'zip': '75015', 'city': 'Paris', 'country': 'France', 'facility': 'Hopital Necker - 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