[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100307049":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":17,"centralContacts":17,"locations":27,"responsibleParty":302,"collaborators":17,"id":304,"slug":305,"hasResults":306,"nctId":307,"briefTitle":308,"officialTitle":309,"acronym":17,"eligibilityCriteria":310,"healthyVolunteers":311,"sex":312,"minAge":313,"maxAge":314,"enrollmentInfo":315,"targetDuration":17,"studyType":318,"phases":319,"briefSummary":321,"conditions":322,"keywords":17,"overallStatus":324,"whyStopped":17,"lastUpdateSubmitDate":325,"lastUpdatePostDateStruct":326,"startDateStruct":328,"completionDateStruct":330,"leadSponsor":332,"locationsCount":333},{"fullName":5,"class":6},"Vertex Pharmaceuticals Incorporated","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Ivacaftor Arm","EXPERIMENTAL","Participants less than (\\\u003C) 24 months of age and weighing 5 to less than (\\\u003C) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to \\\u003C14 kg received 50 mg IVA q12h, and those weighing 14 to \\\u003C25 kg received 75 mg IVA q12h.\n\nParticipants more than or equal (\\>=) 24 months of age and weighing \\\u003C14 kg received 50 mg IVA q12h, and those weighing more than or equal to (\\>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks.\n\nDoses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight.",[13],"Drug: IVA",{"label":15,"type":16,"description":17,"interventionNames":17},"Observational Arm","NO_INTERVENTION",null,[19],{"type":20,"name":21,"description":22,"armGroupLabels":23,"otherNames":24},"DRUG","IVA","Granules for oral administration.",[9],[25,26],"VX-770","ivacaftor",[28,41,51,61,71,81,91,101,111,121,131,141,151,161,171,181,191,201,211,222,232,242,247,255,265,273,281,286,294],{"facility":29,"status":17,"city":30,"state":31,"zip":32,"country":33,"countryCode":34,"cosmosGeoPoint":35,"geoPoint":40,"contacts":17},"University of Alabama at Birmingham","Birmingham","Alabama","35233","United States","US",{"type":36,"coordinates":37},"Point",[38,39],-86.80249,33.52066,{"lat":39,"lon":38},{"facility":42,"status":17,"city":43,"state":44,"zip":45,"country":33,"countryCode":34,"cosmosGeoPoint":46,"geoPoint":50,"contacts":17},"Stanford University","Palo Alto","California","94034",{"type":36,"coordinates":47},[48,49],-122.14302,37.44188,{"lat":49,"lon":48},{"facility":52,"status":17,"city":53,"state":54,"zip":55,"country":33,"countryCode":34,"cosmosGeoPoint":56,"geoPoint":60,"contacts":17},"Alfred I DuPont Hospital for Children","Wilmington","Delaware","19803",{"type":36,"coordinates":57},[58,59],-75.54659,39.74595,{"lat":59,"lon":58},{"facility":62,"status":17,"city":63,"state":64,"zip":65,"country":33,"countryCode":34,"cosmosGeoPoint":66,"geoPoint":70,"contacts":17},"Nemours Children's Hospital","Orlando","Florida","32827",{"type":36,"coordinates":67},[68,69],-81.37924,28.53834,{"lat":69,"lon":68},{"facility":72,"status":17,"city":73,"state":74,"zip":75,"country":33,"countryCode":34,"cosmosGeoPoint":76,"geoPoint":80,"contacts":17},"Center for Advanced Pediatrics","Atlanta","Georgia","30329",{"type":36,"coordinates":77},[78,79],-84.38798,33.749,{"lat":79,"lon":78},{"facility":82,"status":17,"city":83,"state":84,"zip":85,"country":33,"countryCode":34,"cosmosGeoPoint":86,"geoPoint":90,"contacts":17},"Northwestern University","Chicago","Illinois","60611",{"type":36,"coordinates":87},[88,89],-87.65005,41.85003,{"lat":89,"lon":88},{"facility":92,"status":17,"city":93,"state":94,"zip":95,"country":33,"countryCode":34,"cosmosGeoPoint":96,"geoPoint":100,"contacts":17},"Riley Hospital for Children at Indiana University Health","Indianapolis","Indiana","46202",{"type":36,"coordinates":97},[98,99],-86.15804,39.76838,{"lat":99,"lon":98},{"facility":102,"status":17,"city":103,"state":104,"zip":105,"country":33,"countryCode":34,"cosmosGeoPoint":106,"geoPoint":110,"contacts":17},"John Hopkins Hospital","Baltimore","Maryland","21287",{"type":36,"coordinates":107},[108,109],-76.61219,39.29038,{"lat":109,"lon":108},{"facility":112,"status":17,"city":113,"state":114,"zip":115,"country":33,"countryCode":34,"cosmosGeoPoint":116,"geoPoint":120,"contacts":17},"Boston Children's Hospital","Boston","Massachusetts","02115",{"type":36,"coordinates":117},[118,119],-71.05977,42.35843,{"lat":119,"lon":118},{"facility":122,"status":17,"city":123,"state":124,"zip":125,"country":33,"countryCode":34,"cosmosGeoPoint":126,"geoPoint":130,"contacts":17},"Childrens's Hospitals and Clinics of Minnnesota","Minneapolis","Minnesota","55404",{"type":36,"coordinates":127},[128,129],-93.26384,44.97997,{"lat":129,"lon":128},{"facility":132,"status":17,"city":133,"state":134,"zip":135,"country":33,"countryCode":34,"cosmosGeoPoint":136,"geoPoint":140,"contacts":17},"Children's Mercy Hospital","Kansas City","Missouri","64108",{"type":36,"coordinates":137},[138,139],-94.57857,39.09973,{"lat":139,"lon":138},{"facility":142,"status":17,"city":143,"state":144,"zip":145,"country":33,"countryCode":34,"cosmosGeoPoint":146,"geoPoint":150,"contacts":17},"Billings Clinic Hospital","Billings","Montana","59101",{"type":36,"coordinates":147},[148,149],-108.50069,45.78329,{"lat":149,"lon":148},{"facility":152,"status":17,"city":153,"state":154,"zip":155,"country":33,"countryCode":34,"cosmosGeoPoint":156,"geoPoint":160,"contacts":17},"Nationwide Children's Hospital","Columbus","Ohio","43205",{"type":36,"coordinates":157},[158,159],-82.99879,39.96118,{"lat":159,"lon":158},{"facility":162,"status":17,"city":163,"state":164,"zip":165,"country":33,"countryCode":34,"cosmosGeoPoint":166,"geoPoint":170,"contacts":17},"Children's Hospital of Philadelphia","Philadelphia","Pennsylvania","19104",{"type":36,"coordinates":167},[168,169],-75.16362,39.95238,{"lat":169,"lon":168},{"facility":172,"status":17,"city":173,"state":174,"zip":175,"country":33,"countryCode":34,"cosmosGeoPoint":176,"geoPoint":180,"contacts":17},"Texas Children's Hospital","Houston","Texas","77030",{"type":36,"coordinates":177},[178,179],-95.36327,29.76328,{"lat":179,"lon":178},{"facility":182,"status":17,"city":183,"state":184,"zip":185,"country":33,"countryCode":34,"cosmosGeoPoint":186,"geoPoint":190,"contacts":17},"Seattle Children's Hospital","Seattle","Washington","98105",{"type":36,"coordinates":187},[188,189],-122.33207,47.60621,{"lat":189,"lon":188},{"facility":192,"status":17,"city":193,"state":194,"zip":195,"country":33,"countryCode":34,"cosmosGeoPoint":196,"geoPoint":200,"contacts":17},"University of Wisconsin","Madison","Wisconsin","53792",{"type":36,"coordinates":197},[198,199],-89.40123,43.07305,{"lat":199,"lon":198},{"facility":202,"status":17,"city":203,"state":17,"zip":17,"country":204,"countryCode":205,"cosmosGeoPoint":206,"geoPoint":210,"contacts":17},"Queensland Children's Hospital","South Brisbane","Australia","AU",{"type":36,"coordinates":207},[208,209],153.02049,-27.48034,{"lat":209,"lon":208},{"facility":212,"status":17,"city":213,"state":214,"zip":17,"country":215,"countryCode":216,"cosmosGeoPoint":217,"geoPoint":221,"contacts":17},"The Hospital for Sick Children","Toronto","Ontario","Canada","CA",{"type":36,"coordinates":218},[219,220],-79.39864,43.70643,{"lat":220,"lon":219},{"facility":223,"status":17,"city":224,"state":17,"zip":17,"country":225,"countryCode":226,"cosmosGeoPoint":227,"geoPoint":231,"contacts":17},"Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin","Heidelberg","Germany","DE",{"type":36,"coordinates":228},[229,230],8.69079,49.40768,{"lat":230,"lon":229},{"facility":233,"status":17,"city":234,"state":17,"zip":17,"country":235,"countryCode":236,"cosmosGeoPoint":237,"geoPoint":241,"contacts":17},"Children's Health Ireland at Crumlin","Dublin","Ireland","IE",{"type":36,"coordinates":238},[239,240],-6.24889,53.33306,{"lat":240,"lon":239},{"facility":243,"status":17,"city":234,"state":17,"zip":17,"country":235,"countryCode":236,"cosmosGeoPoint":244,"geoPoint":246,"contacts":17},"Children's University Hospital Temple Street",{"type":36,"coordinates":245},[239,240],{"lat":240,"lon":239},{"facility":248,"status":17,"city":249,"state":17,"zip":17,"country":235,"countryCode":236,"cosmosGeoPoint":250,"geoPoint":254,"contacts":17},"University Hospital Limerick","Limerick",{"type":36,"coordinates":251},[252,253],-8.62306,52.66472,{"lat":253,"lon":252},{"facility":256,"status":17,"city":257,"state":17,"zip":17,"country":258,"countryCode":259,"cosmosGeoPoint":260,"geoPoint":264,"contacts":17},"Paediatric Clinical Research Facility","Edinburgh","United Kingdom","UK",{"type":36,"coordinates":261},[262,263],-3.19648,55.95206,{"lat":263,"lon":262},{"facility":266,"status":17,"city":267,"state":17,"zip":17,"country":258,"countryCode":259,"cosmosGeoPoint":268,"geoPoint":272,"contacts":17},"Alder Hey Children's Alder Hey Children's NHS Foundation Trust","Liverpool",{"type":36,"coordinates":269},[270,271],-2.97794,53.41058,{"lat":271,"lon":270},{"facility":274,"status":17,"city":275,"state":17,"zip":17,"country":258,"countryCode":259,"cosmosGeoPoint":276,"geoPoint":280,"contacts":17},"Great Ormond Street Hospital for Sick Children","London",{"type":36,"coordinates":277},[278,279],-0.12574,51.50853,{"lat":279,"lon":278},{"facility":282,"status":17,"city":275,"state":17,"zip":17,"country":258,"countryCode":259,"cosmosGeoPoint":283,"geoPoint":285,"contacts":17},"Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital",{"type":36,"coordinates":284},[278,279],{"lat":279,"lon":278},{"facility":287,"status":17,"city":288,"state":17,"zip":17,"country":258,"countryCode":259,"cosmosGeoPoint":289,"geoPoint":293,"contacts":17},"Royal Manchester Children's Hospital","Manchester",{"type":36,"coordinates":290},[291,292],-2.23743,53.48095,{"lat":292,"lon":291},{"facility":295,"status":17,"city":296,"state":17,"zip":17,"country":258,"countryCode":259,"cosmosGeoPoint":297,"geoPoint":301,"contacts":17},"Oxford University Hospitals NHS Trust, John Radcliffe Hospital","Oxford",{"type":36,"coordinates":298},[299,300],-1.25596,51.75222,{"lat":300,"lon":299},{"type":303,"investigatorFullName":17,"investigatorTitle":17,"investigatorAffiliation":17,"oldNameTitle":17,"oldOrganization":17},"SPONSOR","100307049","phase-3-a-study-to-evaluate-the-safety-of-long-term-ivacaftor-treatment-in-participants-with-cystic-fibrosis-who-are-less-than-24-months-of-age-at-treatment-initiation-and-have-an-approved-ivacaftor-responsive-mutation-100307049",true,"NCT03277196","A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation","A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation","Inclusion Criteria:\n\nIvacaftor Arm: Participants From Study 124 (NCT02725567 ) Part B:\n\n* Participants transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.\n* As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).\n\nIvacaftor Arm: Participants Not From Study 124 Part B:\n\n* Confirmed diagnosis of CF, or 2 CF-causing mutations.\n* An ivacaftor- responsive CFTR mutation on at least 1 allele. Participants will be eligible in countries\u002Fregions where ivacaftor is approved for use in participants 2 years of age and older.\n* As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.\n\nObservational Arm:\n\n* Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.\n\nExclusion Criteria:\n\nIvacaftor Arm: Participants From Study 124 Part B:\n\n* History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant.\n* Participants receiving commercially available ivacaftor treatment\n\nIvacaftor Arm: Participants Not From Study 124 Part B:\n\n* History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant\n* An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1\n* Abnormal liver function at screening\n* Hemoglobin \\\u003C9.5 g\u002FdL at screening\n* History of solid organ or hematological transplantation\n* Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1\n\nObservational Arm:\n\n* Receiving ivacaftor treatment\n\nOther protocol defined Inclusion\u002FExclusion criteria may apply.",false,"ALL","0 Months","24 Months",{"count":316,"type":317},86,"ACTUAL","INTERVENTIONAL",[320],"PHASE3","This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are \\\u003C24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.",[323],"Cystic Fibrosis","COMPLETED","2024-10-01",{"date":327,"type":317},"2024-10-23",{"date":329,"type":317},"2017-08-16",{"date":331,"type":317},"2023-10-02",{"name":5,"class":6},29]