Status:

COMPLETED

Safety Study of Ivacaftor in Subjects With Cystic Fibrosis

Lead Sponsor:

Vertex Pharmaceuticals Incorporated

Collaborating Sponsors:

Cystic Fibrosis Foundation

Conditions:

Cystic Fibrosis

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief Summary

The purpose of this study was to evaluate the safety and tolerability of ivacaftor in patients with cystic fibrosis (CF) who were aged 18 years or older and have a G551D mutation in the cystic fibrosi...

Detailed Description

This was a double-blind, placebo-controlled, cross-over, multiple dose study of up to 28 days of dosing, in subjects with cystic fibrosis (CF) who have a G551D-CTFR gene mutation. Enrollment of 39 sub...

Eligibility Criteria

Inclusion

  • Weighing at least 40 kg
  • Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
  • Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for age, gender, and height
  • Willing to remain on stable medication regimen for the duration of study participation
  • No significant clinical laboratory abnormalities, not pregnant, and willing to use at least 2 highly effective birth control methods during Part 1 and 1 highly effective birth control method during Part 2 of 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
  • Ongoing acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 14 days of Day 1 of the study
  • History of alcohol, medication or illicit drug abuse within one year prior to Day 1
  • Abnormal liver function ≥ 3x the upper limit of normal
  • History of abnormal renal function (creatinine clearance \< 50 mL/min using Cockcroft-Gault equation)
  • History of solid organ or hematological transplantation
  • Pregnant or breast-feeding (for women)
  • Ongoing participation in another therapeutic clinical trial, or prior participation in an investigational drug study without appropriate washout
  • Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)

Key Trial Info

Start Date :

May 1 2007

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

August 1 2008

Estimated Enrollment :

39 Patients enrolled

Trial Details

Trial ID

NCT00457821

Start Date

May 1 2007

End Date

August 1 2008

Last Update

October 5 2012

Active Locations (15)

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Page 1 of 4 (15 locations)

1

University of Alabama Hospital

Birmingham, Alabama, United States, 35211

2

Stanford University Medical Center

Palo Alto, California, United States, 34304

3

The Children's Hospital

Aurora, Colorado, United States, 80045

4

Roy J. and Lucille A. Carver College of Medicine, The University of Iowa

Iowa City, Iowa, United States, 52242-1083