Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Vertex Pharmaceuticals Incorporated
Age
6–11
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
Clareo Health | Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
