An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis

Vertex Pharmaceuticals Incorporated95 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Cystic Fibrosis

Interventions

Ophthalmologic examinationsOTHER

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.

Ivacaftor ExposedDRUG

Eligibility

Sex: ALLMin age: 6 YearsMax age: 11 Years

Medical Language ↔ Plain English

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

Locations (38)

Outcomes

Primary Outcomes

Cataracts (lens opacities)

Time frame: Through Month 24

Best corrected distance vision

Time frame: Through Month 24

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.