{"id":"lum-iva","safety":{"commonSideEffects":[{"rate":"20–30","effect":"Nausea"},{"rate":"15–25","effect":"Diarrhea"},{"rate":"10–20","effect":"Fatigue"},{"rate":"10–15","effect":"Abdominal pain"},{"rate":"10–15","effect":"Headache"},{"rate":"5–10","effect":"Elevated liver transaminases"}]},"_chembl":{"chemblId":"CHEMBL6013288","moleculeType":null,"molecularWeight":"482.57"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Lumacaftor corrects the misfolding of F508del-CFTR protein, allowing it to traffic properly to the cell surface. Ivacaftor then acts as a potentiator, increasing the open probability of the CFTR channel at the cell membrane, enhancing chloride ion transport. 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Incorporated","startDate":"2020-02-24","conditions":"Cystic Fibrosis","enrollment":52},{"nctId":"NCT03601637","phase":"PHASE3","title":"Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del","status":"COMPLETED","sponsor":"Vertex Pharmaceuticals Incorporated","startDate":"2018-09-07","conditions":"Cystic Fibrosis","enrollment":61},{"nctId":"NCT03625466","phase":"PHASE2","title":"A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del","status":"COMPLETED","sponsor":"Vertex Pharmaceuticals Incorporated","startDate":"2018-08-10","conditions":"Cystic Fibrosis","enrollment":51},{"nctId":"NCT02544451","phase":"PHASE3","title":"Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor","status":"COMPLETED","sponsor":"Vertex Pharmaceuticals 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defective cystic fibrosis transmembrane conductance regulator (CFTR) protein. Used for Cystic fibrosis in patients aged 6 years and older who are homozygous for the F508del-CFTR mutation or heterozygous for the F508del-CFTR mutation and one residual function mutation.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}