Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)

Study Overview
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Brief Summary
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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).

Official Title
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Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: ivacaftor


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: ivacaftor|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Mortality: Incidence and cause of deaths |  | 48 Months | 
| Organ transplantation: Incidence and reason for organ transplantations |  | 48 Months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic Fibrosis