Status:
COMPLETED
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborating Sponsors:
Cystic Fibrosis Foundation
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
6-11 years
Phase:
PHASE3
Brief Summary
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane cond...
Detailed Description
This is a Phase 3, 2-part, randomized, double-blind, placebo-controlled, parallel group multicenter study of orally administered ivacaftor in subjects with cystic fibrosis (CF) 6 to 11 years of age wh...
Eligibility Criteria
Inclusion
- Weighing at least 15 kg
- Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
- Forced expiratory volume in 1 second (FEV1) of 40% to 105% (inclusive) of predicted normal for age, gender, and height (Knudson standards) at Screening
- Able to swallow tablets
- As judged by the investigator, parent or legal guardian and subject must have been able to understand protocol requirements, restrictions, and instructions, and the parent or legal guardian should have been able to ensure that the subject complied with, and was likely to complete, the study as planned
- Parent or legal guardian must have signed the informed consent form and corresponding assent must be obtained from the subject
- Willing to use at least 1 highly effective birth control method during the study
- No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
Exclusion
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
- Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
- Abnormal liver function ≥ 3x the upper limit of normal
- Abnormal renal function at Screening
- History of solid organ or hematological transplantation
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
- Use of inhaled hypertonic saline treatment
- Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)
Key Trial Info
Start Date :
August 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2011
Estimated Enrollment :
52 Patients enrolled
Trial Details
Trial ID
NCT00909727
Start Date
August 1 2009
End Date
April 1 2011
Last Update
August 21 2012
Active Locations (29)
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1
University of Alabama
Birmingham, Alabama, United States, 35233-1711
2
Emory Cystic Fibrosis Center
Atlanta, Georgia, United States, 30322
3
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
4
The Cystic Fibrosis Center of Chicago
Glenview, Illinois, United States, 60025