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

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

6 year–11 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Birmingham, Alabama, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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.
Subject must reside in the US and be receiving or planning to receive commercially-available ivacaftor.

Exclusion criteria

Subject is enrolled in an ivacaftor clinical study in which ivacaftor exposure is mandated by the protocol.
Subject has received surgery for cataracts

Treatment and study plan

Ophthalmologic examinations

Other

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 Exposed

Drug

Primary outcomes

Cataracts (lens opacities)

Time frame: Through Month 24
Best corrected distance vision

Time frame: Through Month 24

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Collaborators

Cystic Fibrosis Foundation

Registry information

Important dates

Study start: 2013
Primary completion: 2016
Study completion: 2016
First posted: May 27, 2013
Registry last updated: Aug 23, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed