The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
Name: VX-561
Description: VX-561 tablets for oral administration.Type: DrugVX-561
Name: IVA
Description: 150-mg film-coated tablet for oral administration.Type: DrugIVA
Name: Placebo
Description: Placebo matched to IVA.Type: DrugVX-561
Name: Placebo
Description: Placebos matched to VX-561.Type: DrugIVA
Allocation: Randomized
Parallel Assignment
There are 9 SNPs
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R --- --- S549N ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R ---
Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Key Inclusion Criteria: - Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D - On ivacaftor therapy - FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: - History of clinically significant cirrhosis with or without portal hypertension - History of solid organ or hematological transplantation - Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/Exclusion criteria may apply Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G551D --- --- G178R --- --- S549N --- --- S549R ---