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

Status:	Completed
Conditions:	Pulmonary
Therapuetic Areas:	Pulmonary / Respiratory Diseases
Healthy:	No
Age Range:	2 - 5
Updated:	11/1/2018
Start Date:	May 2016
End Date:	September 2017

A Phase 3, 2-Part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

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.

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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

We found this trial at

sites

Clinical Trial Facility in Minneapolis Minnesota

Minneapolis, Minnesota 55455

from

Minneapolis, MN