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Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Bu çalışma, 6-11 yaş arası ve F508del-CFTR mutasyonu açısından homozigot olan kistik fibrozis (cystic fibrosis) hastalarında Lumacaftor ile Ivacaftor kombinasyonunu incelemiştir. Araştırmaya, kistik fibrozis tanısı doğrulanmış, en az 15 kg ağırlığında ve F508del-CFTR mutasyonunu iki kopya taşıyan çocuklar dahil edilmiştir. Çalışma Faz 3 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Confirmed diagnosis of CF defined as: with 2 CF-causing mutations, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities * Subjects who weigh ≥15 kg without shoes at Screening Visit * Subjects who are homozygous for the F508del-CFTR mutation * Subjects with percent predicted forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) (Part A) or ≥40% (Part B) at Screening Visit where the predicted values are adjusted for age, sex, and height using the Wang equation * Subjects with stable CF disease and who are willing to remain on stable CF medication regimen * Able to swallow tablets Exclusion Criteria: * History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject * Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 of the study * Abnormal liver function as defined in the protocol at Screening Visit * Abnormal renal function as defined in the protocol at Screening Visit * History of solid organ or hematological transplantation * Ongoing participation in an investigational drug study or prior participation in an investigational drug study within 30 days prior of Screening Visit * History or evidence of lens opacity or cataract at Screening Visit * Colonization with organisms associated with a more rapid decline in pulmonary status at Screening Visit (Part A only) * A standard 12-lead ECG demonstrating QTcF \>450 msec at Screening Visit