To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).
Name: ivacaftor
Type: DrugCohort
There are 9 SNPs
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R --- --- S549N ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N ---
Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D). --- G551D --- --- G178R --- --- S549N --- --- S549R ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R ---
Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Inclusion Criteria: - Male or female with confirmed diagnosis of CF16 - At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D - Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent - Signed ICFs and, where appropriate, signed Assent Form - Able to understand the study requirements and comply with study data collection procedures Exclusion Criteria: - Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment - Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation - History of organ transplantation Cystic Fibrosis Fibrosis Cystic Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R ---