A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation



Status:Completed
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:12 - 65
Updated:4/21/2016
Start Date:April 2013
End Date:April 2014

Use our guide to learn which trials are right for you!

A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

The primary objective of the study was to evaluate the efficacy of lumacaftor in combination
with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF)
who are homozygous for the F508del mutation on the CF transmembrane conductance regulator
(CFTR) gene.

This was a Phase 3, randomized, double-blind, placebo-controlled, parallel-group multicenter
study of orally administered lumacaftor in combination with ivacaftor in participants aged
12 years and older with CF who are homozygous for the F508del-CFTR mutation.

The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1
[first dose of study drug] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7
days after the Week 24 Visit).

Inclusion Criteria:

- Confirmed diagnosis of CF

- Homozygous for the F508del CFTR mutation

- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent
(%) and less than or equal to (=<) 90% of predicted normal for age, sex, and height

- Willing to remain on a stable CF medication regimen through Week 24 or, if
applicable, the Safety Follow up Visit

Exclusion Criteria:

- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 4 weeks before first
dose of study drug

- History of solid organ or hematological transplantation

- History of alcohol or drug abuse in the past year

- Ongoing or prior participation in an investigational drug study (including studies
investigating lumacaftor and/or ivacaftor) within 30 days of screening.

- Use of strong inhibitors, moderate inducers, or strong inducers of Cytochrome P450 3A
(CYP3A) within 14 days before Day 1 of dosing
We found this trial at
47
sites
2481
mi
from 02139
Seattle, WA
Click here to add this to my saved trials
140
mi
from 02139
Albany, NY
Click here to add this to my saved trials
1942
mi
from 02139
Alburquerque, NM
Click here to add this to my saved trials
1755
mi
from 02139
Aurora, CO
Click here to add this to my saved trials
9768
mi
from 02139
Brisbane,
Click here to add this to my saved trials
396
mi
from 02139
Buffalo, NY
Click here to add this to my saved trials
Charleston, South Carolina 29412
822
mi
from 02139
Charleston, SC
Click here to add this to my saved trials
547
mi
from 02139
Cleveland, OH
Click here to add this to my saved trials
639
mi
from 02139
Columbus, OH
Click here to add this to my saved trials
1546
mi
from 02139
Dallas, TX
Click here to add this to my saved trials
609
mi
from 02139
Detroit, MI
Click here to add this to my saved trials
1574
mi
from 02139
Fort Worth, TX
Click here to add this to my saved trials
739
mi
from 02139
Grand Rapids, MI
Click here to add this to my saved trials
90
mi
from 02139
Hartford, CT
Click here to add this to my saved trials
1601
mi
from 02139
Houston, TX
Click here to add this to my saved trials
802
mi
from 02139
Indianapolis, IN
Click here to add this to my saved trials
1047
mi
from 02139
Iowa City, IA
Click here to add this to my saved trials
1251
mi
from 02139
Jackson, MS
Click here to add this to my saved trials
1015
mi
from 02139
Jacksonville, FL
Click here to add this to my saved trials
1247
mi
from 02139
Kansas City, KS
Click here to add this to my saved trials
766
mi
from 02139
Lexington, KY
Click here to add this to my saved trials
1132
mi
from 02139
Memphis, TN
Click here to add this to my saved trials
1256
mi
from 02139
Miami, FL
Click here to add this to my saved trials
859
mi
from 02139
Milwaukee, WI
Click here to add this to my saved trials
1117
mi
from 02139
Minneapolis, MN
Click here to add this to my saved trials
205
mi
from 02139
Morristown, NJ
Click here to add this to my saved trials
939
mi
from 02139
Nashville, TN
Click here to add this to my saved trials
New Brunswick, New Jersey 08901
215
mi
from 02139
New Brunswick, NJ
Click here to add this to my saved trials
118
mi
from 02139
New Haven, CT
Click here to add this to my saved trials
180
mi
from 02139
New York, NY
Click here to add this to my saved trials
2682
mi
from 02139
Oakland, CA
Click here to add this to my saved trials
1490
mi
from 02139
Oklahoma City, OK
Click here to add this to my saved trials
1276
mi
from 02139
Omaha, NE
Click here to add this to my saved trials
268
mi
from 02139
Philadelphia, PA
Click here to add this to my saved trials
479
mi
from 02139
Pittsburgh, PA
Click here to add this to my saved trials
99
mi
from 02139
Portland, ME
Click here to add this to my saved trials
469
mi
from 02139
Richmond, VA
Click here to add this to my saved trials
334
mi
from 02139
Rochester, NY
Click here to add this to my saved trials
2617
mi
from 02139
Sacramento, CA
Click here to add this to my saved trials
2091
mi
from 02139
Salt Lake City, UT
Click here to add this to my saved trials
1769
mi
from 02139
San Antonio, TX
Click here to add this to my saved trials
1293
mi
from 02139
Sioux Falls, SD
Click here to add this to my saved trials
2256
mi
from 02139
Spokane, WA
Click here to add this to my saved trials
1034
mi
from 02139
St. Louis, MO
Click here to add this to my saved trials
260
mi
from 02139
Syracuse, NY
Click here to add this to my saved trials
639
mi
from 02139
Toledo, OH
Click here to add this to my saved trials
36
mi
from 02139
Worcester, MA
Click here to add this to my saved trials