Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

Completed · Phase 3 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the 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

NCT00909532

Run by

Vertex Pharmaceuticals Incorporated

People needed

167

Starts

2009-06-01

Expected to finish

2012-11-01

Last updated by the study team

2013-01-18

Who can join

Age: 12 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
• Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
• No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
• Willing to use highly effective birth control methods during the study

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
• Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks 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
• Abnormal renal function at Screening
• History of solid organ or hematological transplantation
• Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
• 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)

Where it is running

• Kaiser Permanente Medical Care Program — Oakland, California, United States
• Cystic Fibrosis Research Office, Stanford University — Palo Alto, California, United States
• Rady Children's Hospital — San Diego, California, United States
• National Jewish Medical and Research Center — Denver, Colorado, United States
• Emory Cystic Fibrosis Center — Atlanta, Georgia, United States
• St. Luke's CF Clinic — Boise, Idaho, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• Indiana University — Indianapolis, Indiana, United States
• University of Iowa — Iowa City, Iowa, United States
• Johns Hopkins University — Baltimore, Maryland, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• Pulmonary, Allergy & Critical Care Medicine, University of Minnesota — Minneapolis, Minnesota, United States
• The Children's Mercy Hospital — Kansas City, Missouri, United States
• Washington University — St Louis, Missouri, United States
• Adult Pulmonary/ CF, University of Nebraska Medical Center — Omaha, Nebraska, United States
• Monmouth Medical Center — Long Branch, New Jersey, United States
• Women and Children's Hospital of Buffalo — Buffalo, New York, United States
• Long Island Jewish Medical Center — New Hyde Park, New York, United States
• SUNY Upstate Medical University — Syracuse, New York, United States
• University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Pediatric & Pulmonary Division, Rainbow Babies/Case Western — Cleveland, Ohio, United States
• University of Alabama — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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