SNPMiner Trials by Shray Alag


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Report for Clinical Trial NCT02445053

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

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).

NCT02445053 Cystic Fibrosis
MeSH: Cystic Fibrosis Fibrosis

1 Interventions

Name: ivacaftor

Type: Drug


Primary Outcomes

Measure: Pulmonary exacerbations - Number of pulmonary exacerbations and duration of treatment for pulmonary exacerbations during Kalydeco treatment compared to the period before Kalydeco treatment

Time: 48 Months

Measure: Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment

Time: 48 Months

Measure: Respiratory Microbiology - Percentage of patients with cultures positive for bacteria other than Pseudomonas aeruginosa and for fungi during Kalydeco treatment compared to the period before Kalydeco treatment

Time: 48 Months

Measure: FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment

Time: 48 Months

Measure: Measures of nutritional status: Absolute change in weight, weight-for-age Z score, body mass index (BMI), and BMI-for-age Z-score during Kalydeco treatment

Time: 48 Months

Measure: Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment

Time: 48 Months

Measure: Mortality: Incidence and cause of deaths

Time: 48 Months

Measure: Organ transplantation: Incidence and reason for organ transplantations

Time: 48 Months

Other Outcomes

Measure: To explore the effect of Kalydeco treatment on Health-Related Quality of Life (HRQoL) in patients with CF and in caregivers of pediatric patients enrolled in the study.

Time: 48 Months

Time Perspective: Prospective

Cohort


There are 9 SNPs

SNPs


1 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 ---

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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E ---


2 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 --- --- 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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D ---


3 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 --- --- 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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R ---


4 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 ---


5 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 ---

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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S ---


6 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 ---

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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N ---


7 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 --- --- 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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P ---


8 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 ---

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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N ---


9 S549R

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 Cystic Fibrosis 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 Cystic Fibrosis Fibrosis null --- G178R --- --- S549N --- --- S549R --- --- G551S --- --- G1244E --- --- S1251N --- --- S1255P --- --- G1349D --- --- G178R --- --- S549N --- --- S549R ---



HPO Nodes