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

Cystic Fibrosis Clinical Trial

— VOCAL  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02445053
• Other study ID #: VX14-770-116
• Secondary ID: 2014-002704-24
• Status: Completed
• Start date: April 2015
• Est. completion date: October 2020

Study information

• Verified date: November 2020
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 75
• Est. completion date: October 2020
• Est. primary completion date: October 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ivacaftor

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Italy,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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.		48 Months
Primary	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
Primary	Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment		48 Months
Primary	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
Primary	FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment		48 Months
Primary	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
Primary	Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment		48 Months
Primary	Mortality: Incidence and cause of deaths		48 Months
Primary	Organ transplantation: Incidence and reason for organ transplantations		48 Months