Clinical trial NCT02445053: Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
Official title: Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2015-05-15
Enrollment: 75
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male and female patients with CF, age 6 years or older, who have 1 of the following CFTR mutations on at least 1 allele: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D

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).


Eligibility 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

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
timeFrame: 48 Months
measure: Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment
timeFrame: 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
timeFrame: 48 Months
measure: FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment
timeFrame: 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
timeFrame: 48 Months
measure: Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment
timeFrame: 48 Months
measure: Mortality: Incidence and cause of deaths
timeFrame: 48 Months
measure: Organ transplantation: Incidence and reason for organ transplantations
timeFrame: 48 Months
Secondary outcomes:
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.
timeFrame: 48 Months
Locations:
city: Bari
country: Italy
city: Messina
country: Italy
city: Milano
country: Italy
city: Milan
country: Italy
city: Naples
country: Italy
city: Potenza
country: Italy
city: Verona
country: Italy
city: Den Haag
country: Netherlands
state: South Holland
city: Amsterdam
country: Netherlands
city: Gronningen
country: Netherlands
city: Heidelberglaan
country: Netherlands
city: Nijmegen
country: Netherlands
city: Rotterdam
country: Netherlands
city: London
country: United Kingdom
state: Greater London
city: Birmingham
country: United Kingdom
state: West Midlands
Study officials:
References: