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

• Sponsor: Vertex Pharmaceuticals Incorporated (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02445053
• Collaborator: (none)
• Enrollment: 75
• Locations: 15
• Actual Duration (Months): 66
• Patients Per Site: 5
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: ivacaftor

Study Design

• Study Type: Observational
• Actual Enrollment: 75 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
• Actual Study Start Date: Apr 1, 2015
• Actual Primary Completion Date: Oct 1, 2020
• Actual Study Completion Date: Oct 1, 2020

Outcome Measures

Primary Outcome Measures

1. 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]

2. Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment [48 Months]

3. 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]

4. FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment [48 Months]

5. 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]

6. Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment [48 Months]

7. Mortality: Incidence and cause of deaths [48 Months]

8. Organ transplantation: Incidence and reason for organ transplantations [48 Months]

Other Outcome Measures

1. 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]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1		Bari		Italy
2		Messina		Italy
3		Milano		Italy
4		Milan		Italy
5		Naples		Italy
6		Potenza		Italy
7		Verona		Italy
8		Den Haag	South Holland	Netherlands
9		Amsterdam		Netherlands
10		Gronningen		Netherlands
11		Heidelberglaan		Netherlands
12		Nijmegen		Netherlands
13		Rotterdam		Netherlands
14		London	Greater London	United Kingdom
15		Birmingham	West Midlands	United Kingdom

Sponsors and Collaborators

• Vertex Pharmaceuticals Incorporated

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Vertex Pharmaceuticals Incorporated
• ClinicalTrials.gov Identifier: NCT02445053
• Other Study ID Numbers: VX14-770-116; 2014-002704-24
• First Posted: May 15, 2015
• Last Update Posted: Nov 13, 2020
• Last Verified: Nov 1, 2020

• Keywords provided by Vertex Pharmaceuticals Incorporated: Cystic Fibrosis
• Additional relevant MeSH terms: Cystic Fibrosis; Fibrosis; Ivacaftor

Study Results

No Results Posted as of Nov 13, 2020