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

Vertex Pharmaceuticals Incorporated75 enrolled

Overview

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).

Study Type

OBSERVATIONAL

Enrollment

Conditions

Cystic Fibrosis

Interventions

ivacaftorDRUG

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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

Locations (15)

Unnamed facility

Bari, Italy

Unnamed facility

Messina, Italy

Unnamed facility

Milan, Italy

Unnamed facility

Milan, Italy

Outcomes

Primary Outcomes

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

Time frame: 48 Months

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

Time frame: 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

Time frame: 48 Months

FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment

Time frame: 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

Time frame: 48 Months

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

Time frame: 48 Months

Mortality: Incidence and cause of deaths

Time frame: 48 Months

Organ transplantation: Incidence and reason for organ transplantations

Time frame: 48 Months

Data from ClinicalTrials.gov

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