Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

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 evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if they meet the eligibility criteria.

Key facts

Study ID

NCT02797132

Run by

Vertex Pharmaceuticals Incorporated

People needed

62

Starts

2016-05-01

Expected to finish

2017-09-01

Last updated by the study team

2018-10-30

Who can join

Age: 2 and older, up to 5. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subjects who weigh ≥8 kilogram (kg) without shoes and wearing light clothing at the Screening Visit
• Subjects with confirmed diagnosis of CF at the Screening Visit
• Subjects who are homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation

You may not qualify if…

• Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1
• A standard 12-lead ECG demonstrating QTc >450 millisecond (msec) at the Screening Visit.
• History of solid organ or hematological transplantation.
• Ongoing or prior participation in an investigational drug study (including studies investigating LUM and/or IVA) within 30 days of the Screening Visit.
• History of cataract/lens opacity or evidence of cataract/lens opacity determined to be clinically significant by a licensed ophthalmologist during the ophthalmologic examination at the Screening Visit

Where it is running

• Study site — Palo Alto, California, United States
• Study site — Aurora, Colorado, United States
• Study site — Chicago, Illinois, United States
• Study site — Indianapolis, Indiana, United States
• Study site — Boston, Massachusetts, United States
• Study site — Minneapolis, Minnesota, United States
• Study site — Kansas City, Missouri, United States
• Study site — Buffalo, New York, United States
• Study site — Chapel Hill, North Carolina, United States
• Study site — Cincinnati, Ohio, United States
• Study site — Cleveland, Ohio, United States
• Study site — Columbus, Ohio, United States
• Study site — Philadelphia, Pennsylvania, United States
• Study site — Charleston, South Carolina, United States
• Study site — Houston, Texas, United States
• Study site — Norfolk, Virginia, United States
• Study site — Seattle, Washington, United States
• Study site — Vancouver, British Columbia, Canada
• Study site — Toronto, Ontario, Canada
• Study site — Montreal, Canada

Full record on ClinicalTrials.gov

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