Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR protein is located on the cell membrane of the airways and sweat ducts. It is a chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in tissues. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel. The increase in CFTR chloride ion channel opening may prevent hyperabsorption of water in the airway tissues, allowing for more favorable maintenance of airway hydration in people with CF.
This study has 2 parts. Part A is a pharmacokinetic evaluation of a single dose of VX-770 to determine the appropriate dose for Part B. Pharmacokinetics looks at how a drug is absorbed, distributed, metabolized, and eliminated by the body. Part B will will look at the effectiveness of VX-770 in the pediatric population aged 6 to 11 years who have CF and the G551D mutation and safety over a longer treatment period.
ELIGIBILITY
Inclusion Criteria:
There are inclusion criteria for Part A and Part B of the study. Please note additional inclusion criteria exist for those who enroll in Part A and choose to go into Part B - contact the participating site for that information.
For Part A of the study:
Male or female
Diagnosis of cystic fibrosis
Have mutation in at least 1 allele of G551D
FEV1% 40% to 90%
Age 6 to 11 years
Weight > 15 kg without shoes at Screening
Females of childbearing potential must have a negative serum pregnancy test at screening and a negative urine test on Part A Day 1
There are exclusion criteria for Part A and Part B of the study. Please note additional exclusion criteria exist for those who enroll in Part A and choose to go into Part B - contact the participating site for that information.
For Part A of the study:
History of any illness 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 subject.
Subjects should not have had an acute upper or lower respiratory infection or pulmonary exacerbation or changes in therapy for pulmonary disease within 4 weeks prior to Part A Day 1.
Pregnant, breast-feeding, or not willing to meet the contraception requirements.
Hemoglobin < 10 g/dL at screening
History of solid organ or hematological transplantation.
For Part B of the study (those only entering in Part B):
History of any illness 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 subject.
Subjects should not have had an acute upper or lower respiratory infection or pulmonary exacerbation or changes in therapy for pulmonary disease within 4 weeks prior to Part A Day 1.
Pregnant, breast-feeding, or not willing to meet the contraception requirements.
Hemoglobin < 10 g/dL at screening
History of solid organ or hematological transplantation
On hypertonic saline treatment ( can be eligible if on hypertonic saline but must stop at least 6 weeks prior to Part B Day 1)
www.clinicaltrials.gov for the most current information
PARTICIPATING SITES:
AL
The Children's Hospital/UAB (Pediatric), Birmingham, AL, 35233
Contact Information:
Heather Young Phone: 205-939-9568 Email: hyoung@peds.uab.edu
IL
Children's Memorial Hospital Northwestern University (Pediatric), Chicago, IL, 60614
Contact Information:
Cathy Powers Phone: 773-880-8223 Email: cpowers@childrensmemorial.org
IN
Riley Hospital for Children Indiana University Medical Center (Pediatric), Indianapolis, IN, 46202
Contact Information:
Terry Barclay Phone: 317-274-3201 Email: tbarclay@iupui.edu
University of Michigan Health System (Pediatric), Ann Arbor, MI, 48109
Contact Information:
Ermelinda Sakmar Phone: 734-936-6352 Email: ermee@umich.edu
Children's Hospital of Michigan (Pediatric), Detroit, MI, 48201
Contact Information:
Catherine Van Wagnen Phone: 313-745-4737 Email: cvanwag@med.wayne.edu
MO
The Children's Mercy Hospital -- University of Missouri at Kansas City (Pediatric and Adult), Kansas City, MO, 64108
Contact Information:
Candy Schmoll Phone: 816-983-6410 Email: cschmoll@cmh.edu
NE
University of Nebraska Medical Center (Pediatric), Omaha, NE, 68198
Contact Information:
Diane Acquazzino Phone: 402-559-4087 Email: dracquaz@unmc.edu
PA
Hershey Medical Center Pennsylvania State University (Pediatric), Hershey, PA, 17033
Contact Information:
Diane Kitch Phone: (717) 531-5646 Email: dkitch@psu.edu
Children's Hospital of Pittsburgh of UPMC - University of Pittsburgh (Pediatric), Pittsburgh, PA, 15213
Contact Information:
Elizabeth Hartigan Phone: 412-692-7060 Email: elizabeth.hartigan@chp.edu
TN
East Tennessee Children's Hospital (Pediatric and Adult), Knoxville, TN, 37916
Contact Information:
Judy Marciel Phone: 865-637-8018 x240 Email: jmarciel@etch.com
UT
Intermountain Cystic Fibrosis Center University of Utah Health Sciences Center (Pediatric), Salt Lake City, UT, 84132
Contact Information:
Jane Vroom Phone: 801-587-7458 Email: jane.vroom@hsc.utah.edu
