Ivacaftor in People with CF age 2 to 5 years with a CFTR Gating Mutation
Study Type:
Interventional
Intervention Category:
CFTR Modulation
Study Sponsor:
Vertex
Study Phase:
3
Recruitment Status:
Recruiting
Study Drug(s):
Kalydeco
Number of Participants Being Recruited:
28
Single / Multi-Center:
Multi-Center
STUDY BACKGROUND INFORMATION:
This is an open-label study designed to look at the safety, pharmacokinetics (PK), pharmacodynamics(PD), and effectiveness of ivacaftor in people with CF who are 2 through 5 years of age and have a CFTR gating mutation in at least 1 allele. Part A is designed to look at the safety and PK of multiple-dose administration of ivacaftor in children 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to look at the safety, PK, PD, and efficacy of ivacaftor in children 2 through 5 years of age.
Ivacaftor is currently approved by the US FDA and the EMA under the trade name Kalydeco® for the treatment of cystic fibrosis in patients 6 years of age and older who have the G551D-CFTR mutation
ELIGIBILITY
Age:
2 Years - 5 Years
FEV1:
Not Applicable
P. aeruginosa status:
Not applicable
B. cepacia status:
Not applicable
Other Primary Eligibility Requirements:
Eligible subjects must have a CFTR Gating Mutation in at least one allele
Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific
trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.
FOR MORE INFORMATION:
Sponsor Contact Information:
Medical Monitor, Vertex (617) 444-6777 medicalinfo@vrtx.com
The sites listed below are currently recruiting subjects. Please note that this list generally lists either the pediatric or adult program but not both even though both programs may be recruiting subjects. Also, this list does not include sites that may be participating in the study but are not yet open for recruitment. If you are interested in this study please contact your center for more information.
AL
The Children's Hospital/University of Alabama at Birmingham (Pediatric), Birmingham, AL, 35233
Contact Information:
Hathorne, Heather Phone: (205) 638-9568 Email: hhathorne@peds.uab.edu
IN
Riley Hospital for Children Indiana University Medical Center (Pediatric), Indianapolis, IN, 46202
Contact Information:
Richardson, Stacey Phone: (317) 948-7121 Email: stlyrich@iupui.edu
Helen DeVos Women and Children's Center (Pediatric), Grand Rapids, MI, 49503
Contact Information:
Symington, Thomas Phone: (616) 391-9366 Email: thomas.symington@spectrumhealth.org
MO
The Children's Mercy Hospital (Pediatric and Adult), Kansas City, MO, 64108
Contact Information:
Schmoll, Candy Phone: (816) 701-1339 Email: cschmoll@cmh.edu
UT
Intermountain Cystic Fibrosis Center (Pediatric), Salt Lake City, UT, 84132
Contact Information:
Vroom, Jane Phone: (801) 587-7458 Email: jane.vroom@hsc.utah.edu
