This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations, compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either double-blind CTP-656 or placebo, or open-label Kalydeco.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
11
University of Southern California
Los Angeles, California, United States
Stanford Hospital
Palo Alto, California, United States
Children's National Health
Washington D.C., District of Columbia, United States
Change From Baseline in Sweat Chloride at Day 28
Time frame: From baseline at Day 28
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Time frame: From baseline at Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28
Time frame: From baseline at Day 28
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University of Miami
Miami, Florida, United States
Rush University
Chicago, Illinois, United States
Indiana University
Indianapolis, Indiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Massachusetts
Worcester, Massachusetts, United States
Washington University
St Louis, Missouri, United States
Atlantic Health
Morristown, New Jersey, United States
...and 4 more locations