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

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis

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).

N.J. Simmonds, Kors van der Ent, C. Colombo, Nils Kinnman, Cynthia DeSouza et al. (8 authors)

2021-11-01

10.1136/thorax-2021-btsabstracts.66

S61 Respiratory microbiology outcomes from an observational study of ivacaftor in people with cystic fibrosis and non-G551D gating mutations (VOCAL)

Carlo Castellani, N.J. Simmonds, C. Colombo, Nils Kinnman, Cynthia DeSouza et al. (9 authors)

2021-11-01

10.1136/thorax-2021-btsabstracts.67

Observational study of ivacaftor in people with cystic fibrosis and selected non-G551D gating mutations: final results from VOCAL

Nicholas J. Simmonds, Kors van der Ent, Carla Colombo, Nils Kinnman, Cynthia DeSouza et al. (8 authors)

2021-09-05

10.1183/13993003.congress-2021.pa2100

Respiratory microbiology outcomes from an observational study of ivacaftor in people with cystic fibrosis and non-G551D gating mutations (VOCAL)

Carlo Castellani, Nicholas J. Simmonds, Carla Colombo, Nils Kinnman, Cynthia DeSouza et al. (9 authors)

2021-09-05

10.1183/13993003.congress-2021.pa2102

S15 An observational study of ivacaftor in patients with cystic fibrosis (CF) and selected non-G551D gating mutations: outcomes from the second interim analysis of the VOCAL study

N.J. Simmonds, Carlo Castellani, Carla Colombo, Kors van der Ent, Lokesh K. Jha et al. (8 authors)

2019-11-12

10.1136/thorax-2019-btsabstracts2019.21

Interventions

DRUGivacaftor

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

6 Years to No maximum

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

Design outcomes

Primary

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

Other

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.	48 Months

Countries

Italy, Netherlands, United Kingdom

Outcome results

None listed