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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02445053
• Date of registration: 11/05/2015
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) VOCAL
• Scientific title: Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
• Date of first enrolment: April 2015
• Target sample size: 75
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02445053
• Study type: Observational

Countries of recruitment

Italy, Netherlands, United Kingdom

Key inclusion & exclusion criteria

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



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: ivacaftor

Primary Outcome(s)

Comorbidities: Incidence and prevalence of comorbidities 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]

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]

Mortality: Incidence and cause of deaths [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]

Organ transplantation: Incidence and reason for organ transplantations [Time Frame: 48 Months]

FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment [Time Frame: 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 [Time Frame: 48 Months]

Secondary ID(s)

2014-002704-24

VX14-770-116

Source(s) of Monetary Support

Please refer to primary and secondary sponsors