Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
Completed
- Conditions
- Cystic Fibrosis
- Registration Number
- NCT02445053
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 75
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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Mortality: Incidence and cause of deaths 48 Months Organ transplantation: Incidence and reason for organ transplantations 48 Months Pulmonary exacerbations - Number of pulmonary exacerbations and duration of treatment for pulmonary exacerbations during Kalydeco treatment compared to the period before Kalydeco treatment 48 Months Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment 48 Months 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 48 Months FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment 48 Months 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 48 Months Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment 48 Months
- Secondary Outcome Measures
Name Time Method