Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

The purpose of this study was to evaluate the safety and efficacy of ivacaftor in participants with cystic fibrosis (CF) who were aged 12 years or older and were homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. 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 adenosine monophosphate (AMP)-dependent protein kinase A (PKA) activation.

Key facts

Study ID

NCT00953706

Run by

Vertex Pharmaceuticals Incorporated

People needed

140

Starts

2009-09-01

Expected to finish

2013-05-01

Last updated by the study team

2015-09-11

Who can join

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

You may qualify if…

• Confirmed diagnosis of cystic fibrosis (CF) and homozygous for F508del-CFTR mutation
• Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for age, gender, and height
• Willing to use at least 2 highly effective birth control methods during the study
• No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
• Able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, 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
• 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 >=3 x the upper limit of normal
• Abnormal renal function at Screening
• History of solid organ or hematological transplantation
• Pregnant or breast-feeding (for women)
• Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to screening
• Previous participation in a VX-809 study
• Used inhaled hypertonic saline treatment
• Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)

Where it is running

• University of Alabama — Birmingham, Alabama, United States
• Providence Medical Center — Anchorage, Alaska, United States
• Kaiser Permanente Medical Care Program — Oakland, California, United States
• Connecticut Children's Medical Center — Hartford, Connecticut, United States
• University of Miami Miller School of Medicine — Miami, Florida, United States
• Nemours Children's Clinic — Orlando, Florida, United States
• St. Luke's CF clinic — Boise, Idaho, United States
• University of Chicago — Chicago, Illinois, United States
• Riley Hospital for Children — Indianapolis, Indiana, United States
• Maine Medical Center — Portland, Maine, United States
• Massachusetts General Hospital — Boston, Massachusetts, United States
• University of Massachussetts Medical School — Worcester, Massachusetts, United States
• Helen DeVos Children's Hospital; Spectrum Health Hospitals — Grand Rapids, Michigan, United States
• The Children's Mercy Hospital — Kansas City, Missouri, United States
• Dartmouth-Hitchcock Medical Center — Lebanon, New Hampshire, United States
• Monmouth Medical Center — Long Branch, New Jersey, United States
• Morristown Memorial Hospital — Morristown, New Jersey, United States
• Albany Medical College — Albany, New York, United States
• Women and Children's Hospital of Buffalo — Buffalo, New York, United States
• New York Medical College — Hawthorne, New York, United States
• The CF Center, Beth Israel Medical Center — New York, New York, United States
• Columbia University Medical Center — New York, New York, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Cincinnati Children's Hospital — Cincinnati, Ohio, United States
• Toldedo Children's Hospital — Toledo, Ohio, United States

Full record on ClinicalTrials.gov

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