Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation



Status:Completed
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:18 - Any
Updated:4/21/2016
Start Date:October 2010
End Date:April 2014

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

A Phase 2, Multicenter, Double-Blinded, Placebo-Controlled, Multiple-Dose Study to Evaluate Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and
pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with
ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the
F508del-CFTR mutation.


Inclusion Criteria:

- Male or female participants with confirmed diagnosis of CF

- Must have the F508del-CFTR mutation on at least 1 allele.

- FEV1 greater than equal (>=) 40% of predicted normal for age, gender, and height
(Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age,
gender, and height (Hankinson standards (Cohort 4)

- Participant of child-bearing potential and who are sexually active must meet the
contraception requirements

Exclusion Criteria:

- History of any illness or condition that, in the opinion of the investigator might
confound the results of the study or pose an additional risk in administering study
drug to the participant (e.g., cirrhosis with portal hypertension).

- An acute illness including acute upper or lower respiratory infection, pulmonary
exacerbation or changes in therapy (including antibiotics) for pulmonary disease
within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first
dose of study drug.

- History of solid organ or hematological transplantation.

- History of alcohol abuse or drug addiction in the past year, including cannabis,
cocaine, and opiates.

- Ongoing participation in another therapeutic clinical study, or prior participation
in an investigational drug study without appropriate washout

- Pregnant or nursing females; females of childbearing potential who are unwilling or
unable to use an acceptable method of non-hormonal contraception

- Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3

- Ongoing or prior participation in an investigational drug study within 30 days of the
Screening Visit

- Heterozygous participants who participated in Cohort 2 and meet the eligibility
criteria for Cohort 4 may participate in Cohort 4

- Evidence of lens opacity or cataract as determined by the ophthalmologic examination
(Cohort 4 only)
We found this trial at
40
sites
1511
mi
from 91732
Minneapolis, MN
Click here to add this to my saved trials
8190
mi
from 91732
Adelaide,
Click here to add this to my saved trials
2040
mi
from 91732
Akron, OH
Click here to add this to my saved trials
2343
mi
from 91732
Anchorage, AK
Click here to add this to my saved trials
1919
mi
from 91732
Atlanta, GA
Click here to add this to my saved trials
2301
mi
from 91732
Baltimore, MD
Click here to add this to my saved trials
1784
mi
from 91732
Birmingham, AL
Click here to add this to my saved trials
665
mi
from 91732
Boise, ID
Click here to add this to my saved trials
2578
mi
from 91732
Boston, MA
Click here to add this to my saved trials
2180
mi
from 91732
Buffalo, NY
Click here to add this to my saved trials
2194
mi
from 91732
Chapel Hill, NC
Click here to add this to my saved trials
Charleston, South Carolina 29412
2184
mi
from 91732
Charleston, SC
Click here to add this to my saved trials
1729
mi
from 91732
Chicago, IL
Click here to add this to my saved trials
2032
mi
from 91732
Cleveland, OH
Click here to add this to my saved trials
1959
mi
from 91732
Columbus, OH
Click here to add this to my saved trials
817
mi
from 91732
Denver, CO
Click here to add this to my saved trials
1528
mi
from 91732
Iowa City, IA
Click here to add this to my saved trials
1339
mi
from 91732
Kansas City, KS
Click here to add this to my saved trials
99
mi
from 91732
La Jolla, CA
Click here to add this to my saved trials
2515
mi
from 91732
Lebanon, NH
Click here to add this to my saved trials
1881
mi
from 91732
Lexington, KY
Click here to add this to my saved trials
21
mi
from 91732
Long Beach, CA
Click here to add this to my saved trials
2321
mi
from 91732
Miami, FL
Click here to add this to my saved trials
2486
mi
from 91732
New Haven, CT
Click here to add this to my saved trials
2449
mi
from 91732
New Hyde Park, NY
Click here to add this to my saved trials
1655
mi
from 91732
New Orleans, LA
Click here to add this to my saved trials
2435
mi
from 91732
New York, NY
Click here to add this to my saved trials
1164
mi
from 91732
Oklahoma City, OK
Click here to add this to my saved trials
1300
mi
from 91732
Omaha, NE
Click here to add this to my saved trials
2182
mi
from 91732
Orlando, FL
Click here to add this to my saved trials
329
mi
from 91732
Palo Alto, CA
Click here to add this to my saved trials
2375
mi
from 91732
Philadelphia, PA
Click here to add this to my saved trials
2119
mi
from 91732
Pittsburg, PA
Click here to add this to my saved trials
2119
mi
from 91732
Pittsburgh, PA
Click here to add this to my saved trials
2245
mi
from 91732
Rochester, NY
Click here to add this to my saved trials
364
mi
from 91732
Sacramento, CA
Click here to add this to my saved trials
1313
mi
from 91732
Sioux Falls, SD
Click here to add this to my saved trials
1572
mi
from 91732
St. Louis, MO
Click here to add this to my saved trials
2319
mi
from 91732
Syracuse, NY
Click here to add this to my saved trials
2136
mi
from 91732
Tampa, FL
Click here to add this to my saved trials