Safety Study of Ivacaftor in Subjects With Cystic Fibrosis

Completed · Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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 fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.

Key facts

Study ID: NCT00457821
Run by: Vertex Pharmaceuticals Incorporated
People needed: 39
Starts: 2007-05-01
Expected to finish: 2008-08-01
Last updated by the study team: 2012-10-05

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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)

Where it is running

University of Alabama Hospital — Birmingham, Alabama, United States
Stanford University Medical Center — Palo Alto, California, United States
The Children's Hospital — Aurora, Colorado, United States
Roy J. and Lucille A. Carver College of Medicine, The University of Iowa — Iowa City, Iowa, United States
Johns Hopkins Hospital — Baltimore, Maryland, United States
Pulmonary and Critical Care Medicine, Massachusetts General Hospital — Boston, Massachusetts, United States
Children's Hospital of Boston — Boston, Massachusetts, United States
Division of Pulmonary, Allergy and Critical Care Medicine, University of Minnesota — Minneapolis, Minnesota, United States
Cystic Fibrosis Pulmonary Research and Treatment Center, University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
Rainbow Babies and Children's Hospital — Cleveland, Ohio, United States
The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
University of Pittsburgh — Pittsburgh, Pennsylvania, United States
Pulmonary Critical Care, University of Washington — Seattle, Washington, United States
Division of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children — Toronto, Ontario, Canada
CF Clinic, Pediatric Pulmonology and Neonatology, Medical School of Hannover — Straße, Hannover, Germany

Full record on ClinicalTrials.gov

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