Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years 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 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective 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

NCT00909727

Run by

Vertex Pharmaceuticals Incorporated

People needed

52

Starts

2009-08-01

Expected to finish

2011-04-01

Last updated by the study team

2012-08-21

Who can join

Age: 6 and older, up to 11. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

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
• 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)

Where it is running

• University of Alabama — Birmingham, Alabama, United States
• Emory Cystic Fibrosis Center — Atlanta, Georgia, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• The Cystic Fibrosis Center of Chicago — Glenview, Illinois, United States
• Riley Hospital for Children — Indianapolis, Indiana, United States
• University of Iowa Department of Pediatrics — Iowa City, Iowa, 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
• Children's Hospital of Michigan — Detroit, Michigan, United States
• Helen DeVos Children's Hospital Spectrum Health Hospitals — Grand Rapids, Michigan, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• The Children's Mercy Hospital — Kansas City, Missouri, United States
• University of Nebraska Medical Center Pediatric Pulmonary/ CF — Omaha, Nebraska, United States
• East Tennessee Children's Hospital Pediatric Pulmonary and Respiratory Care — Knoxville, Tennessee, United States
• University of Utah Pediatric Pulmonology — Salt Lake City, Utah, United States
• University of Virginia Pediatric Respiratory Medicine — Charlottesville, Virginia, United States
• The Children's Hospital Westmead — Westmead, New South Wales, Australia
• Royal Children's Hospital Brisbane — Herston, Queensland, Australia
• Royal Children's Hospital Melbourne — Parkville, Victoria, Australia
• Princess Margaret Hospital for Children — Subiaco, Western Australia, Australia
• British Columbia Children's Hospital — Vancouver, British Columbia, Canada
• Hospital for Sick Children CF Center — Toronto, Ontario, Canada
• Hôpital Robert Debré - Service de gastro-entérologiemucoviscidose et nutrition — Paris, France
• Kinder- und Jugendklinik Universitätsklinikum Erlangen — Erlangen, Germany

Full record on ClinicalTrials.gov

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