Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Completed · Phase 3

Conditions studied: Cystic Fibrosis

In brief

This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study to evaluate the pharmacokinetics, safety, and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the F508del-mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Key facts

Study ID

NCT01897233

Run by

Vertex Pharmaceuticals Incorporated

People needed

62

Starts

2013-07-01

Expected to finish

2015-10-01

Last updated by the study team

2017-06-20

Who can join

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

You may qualify if…

• Confirmed diagnosis of CF defined as: with 2 CF-causing mutations, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
• Subjects who weigh ≥15 kg without shoes at Screening Visit
• Subjects who are homozygous for the F508del-CFTR mutation
• Subjects with percent predicted forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) (Part A) or ≥40% (Part B) at Screening Visit where the predicted values are adjusted for age, sex, and height using the Wang equation
• Subjects with stable CF disease and who are willing to remain on stable CF medication regimen
• Able to swallow tablets

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 28 days before Day 1 of the study
• Abnormal liver function as defined in the protocol at Screening Visit
• Abnormal renal function as defined in the protocol at Screening Visit
• History of solid organ or hematological transplantation
• Ongoing participation in an investigational drug study or prior participation in an investigational drug study within 30 days prior of Screening Visit
• History or evidence of lens opacity or cataract at Screening Visit
• Colonization with organisms associated with a more rapid decline in pulmonary status at Screening Visit (Part A only)
• A standard 12-lead ECG demonstrating QTcF >450 msec at Screening Visit

Where it is running

• Study site — Birmingham, Alabama, United States
• Study site — Tucson, Arizona, United States
• Study site — Long Beach, California, United States
• Study site — Palo Alto, California, United States
• Study site — Aurora, Colorado, United States
• Study site — Atlanta, Georgia, United States
• Study site — Indianapolis, Indiana, United States
• Study site — Boston, Massachusetts, United States
• Study site — Kansas City, Missouri, United States
• Study site — St Louis, Missouri, United States
• Study site — Buffalo, New York, United States
• Study site — Rochester, New York, United States
• Study site — Syracuse, New York, United States
• Study site — Charleston, South Carolina, United States
• Study site — Austin, Texas, United States
• Study site — Salt Lake City, Utah, United States
• Study site — Norfolk, Virginia, United States
• Study site — Seattle, Washington, United States
• Study site — Milwaukee, Wisconsin, United States
• Study site — Toronto, Ontario, Canada

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.