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

Sponsor

Vertex Pharmaceuticals Incorporated (Industry)

Overall Status

Completed

CT.gov ID

NCT01863238

Collaborator

Cystic Fibrosis Foundation (Other)

Enrollment

Locations

Duration (Months)

2.5

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Other: Ophthalmologic examinations Drug: Ivacaftor Exposed

Study Design

Study Type:

Observational

Actual Enrollment :

95 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Study Start Date :

May 1, 2013

Actual Primary Completion Date :

May 1, 2016

Actual Study Completion Date :

May 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Ivacaftor Treated	Other: Ophthalmologic examinations 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. Drug: Ivacaftor Exposed

Arm

Intervention/Treatment

Ivacaftor Treated

Other: Ophthalmologic examinations

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.

Drug: Ivacaftor Exposed

Outcome Measures

Primary Outcome Measures

Cataracts (lens opacities) [Through Month 24]
Best corrected distance vision [Through Month 24]

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 11 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	City	State	Country
1	Birmingham	Alabama	United States
2	Tucson	Arizona	United States
3	Long Beach	California	United States
4	Aurora	Colorado	United States
5	Wilmington	Delaware	United States
6	St. Petersburg	Florida	United States
7	Boise	Idaho	United States
8	Chicago	Illinois	United States
9	Indianapolis	Indiana	United States
10	Kansas City	Kansas	United States
11	Louisville	Kentucky	United States
12	Portland	Maine	United States
13	Boston	Massachusetts	United States
14	Ann Arbor	Michigan	United States
15	Detroit	Michigan	United States
16	Minneapolis	Minnesota	United States
17	Columbia	Missouri	United States
18	Kansas City	Missouri	United States
19	Omaha	Nebraska	United States
20	Rochester	New York	United States
21	Akron	Ohio	United States
22	Cincinnati	Ohio	United States
23	Toledo	Ohio	United States
24	Oklahoma City	Oklahoma	United States
25	Portland	Oregon	United States
26	Philadelphia	Pennsylvania	United States
27	Pittsburgh	Pennsylvania	United States
28	Providence	Rhode Island	United States
29	Charleston	South Carolina	United States
30	Knoxville	Tennessee	United States
31	Nashville	Tennessee	United States
32	Fort Worth	Texas	United States
33	Houston	Texas	United States
34	Salt Lake City	Utah	United States
35	Charlottesville	Virginia	United States
36	Seattle	Washington	United States
37	Spokane	Washington	United States
38	Morgantown	West Virginia	United States

Sponsors and Collaborators

Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Vertex Pharmaceuticals Incorporated

ClinicalTrials.gov Identifier:

NCT01863238

Other Study ID Numbers:

VX12-770-115

First Posted:

May 27, 2013

Last Update Posted:

Aug 23, 2016

Last Verified:

Jul 1, 2015

Keywords provided by Vertex Pharmaceuticals Incorporated

Cystic Fibrosis

Additional relevant MeSH terms:

Cystic Fibrosis

Fibrosis

Ivacaftor

Study Results

No Results Posted as of Aug 23, 2016