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

November 12, 2020 updated by: Vertex Pharmaceuticals Incorporated
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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

75

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Bari, Italy
      • Messina, Italy
      • Milan, Italy
      • Milano, Italy
      • Naples, Italy
      • Potenza, Italy
      • Verona, Italy
  • Netherlands
      • Amsterdam, Netherlands
      • Gronningen, Netherlands
      • Heidelberglaan, Netherlands
      • Nijmegen, Netherlands
      • Rotterdam, Netherlands
    • South Holland
      • Den Haag, South Holland, Netherlands
  • United Kingdom
    • Greater London
      • London, Greater London, United Kingdom
    • West Midlands
      • Birmingham, West Midlands, United Kingdom

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
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
Time Frame: 48 Months
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
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
48 Months
FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment
Time Frame: 48 Months
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
48 Months
Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment
Time Frame: 48 Months
48 Months
Mortality: Incidence and cause of deaths
Time Frame: 48 Months
48 Months
Organ transplantation: Incidence and reason for organ transplantations
Time Frame: 48 Months
48 Months

Other Outcome Measures

Outcome Measure
Time Frame
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.
Time Frame: 48 Months
48 Months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

April 1, 2015

Primary Completion (ACTUAL)

October 1, 2020

Study Completion (ACTUAL)

October 1, 2020

Study Registration Dates

First Submitted

May 11, 2015

First Submitted That Met QC Criteria

May 11, 2015

First Posted (ESTIMATE)

May 15, 2015

Study Record Updates

Last Update Posted (ACTUAL)

November 13, 2020

Last Update Submitted That Met QC Criteria

November 12, 2020

Last Verified

November 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX14-770-116
  • 2014-002704-24 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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