This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
Name: CTP-656, 20 mg (QD)
Type: DrugCTP-656, 20 mg, QD
Name: CTP-656, 100 mg (QD)
Type: DrugCTP-656, 100 mg, QD
Name: CTP-656, 150 mg (QD)
Type: DrugCTP-656, 150 mg, QD
Name: Placebo (QD)
Type: DrugPlacebo, Oral Tablet, QD
Name: Kalydeco, 150 mg Tablet (BID; open-label)
Type: DrugKalydeco, 150 mg Tablet (open label)
Allocation: Randomized
Parallel Assignment
There are 9 SNPs
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N --- --- S1255P ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N --- --- S1255P ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N --- --- S1255P --- --- S549N ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N --- --- S1255P --- --- S549N ---
Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N --- --- S1255P --- --- S549N --- --- S549R ---
Exclusion Criteria: - Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment - Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes - History of hepatitis C or chronic active hepatitis B infection - History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening - Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening - Abnormal liver function - History of abnormal renal function - History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females - History of solid organ or hematological transplantation - Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening - Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose Inclusion Criteria: - 18 years of age or older - Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R. --- G551D --- --- G1244E --- --- G1349D --- --- G178R --- --- G551S --- --- S1251N --- --- S1255P --- --- S549N --- --- S549R ---