The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation
Name: ivacaftor
Allocation: Non-Randomized
Single Group Assignment
There are 9 SNPs
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R --- --- S549N ---
- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. --- G551D --- --- G178R --- --- S549N --- --- S549R ---