Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis
Brief summary
This study is designed to evaluate the risk of cataracts (lens opacities) and describe the best corrected distance vision (with glasses/contacts for those who wear them) of pediatric patients with Cystic Fibrosis who are 11 years of age or younger at the time of ivacaftor treatment initiation and are receiving or planning to receive commercially-available ivacaftor in the US.
Interventions
Subjects will undergo an ophthalmologic examination at study entry (Day 1) and every 6 months thereafter for 2 years (Months 6, 12, 18, and 24). Subjects who discontinue treatment with commercially-available ivacaftor for any reason and are willing to continue participation in the study will complete the study.
DRUGIvacaftor Exposed
Sponsors
Cystic Fibrosis Foundation
Vertex Pharmaceuticals Incorporated
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
6 Years to 11 Years
Healthy volunteers
No
Inclusion criteria
1. Subject must be 11 years of age or younger at the time of treatment initiation with ivacaftor (as part of clinical trial) or commercially-available ivacaftor. 2. Subject must reside in the US and be receiving or planning to receive commercially-available ivacaftor.
Exclusion criteria
1. Subject is enrolled in an ivacaftor clinical study in which ivacaftor exposure is mandated by the protocol. 2. Subject has received surgery for cataracts
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Cataracts (lens opacities) | Through Month 24 |
| Best corrected distance vision | Through Month 24 |
Countries
United States
Outcome results
None listed