Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
- Registration Number
- NCT02971839
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 11
- 18 years of age or older
- Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R.
- Has been stable on Kalydeco therapy for at least 3 months prior to screening
- Has FEV1 ≥ 60% of predicted normal for age, sex, and height at screening and baseline (Day 1) assessments
- Weighs at least 40 kg at screening
- Patients of either gender and women of childbearing potential must be willing to use a medically highly effective form of birth control during the treatment period and 30 days after the last dose of study treatment.
- Acute upper respiratory infection or lower respiratory infection, pulmonary exacerbation, or changes in therapy within 4 weeks of study treatment
- Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes
- History of hepatitis C or chronic active hepatitis B infection
- History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2 years prior to screening
- Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within 2 years prior to Screening
- Abnormal liver function
- History of abnormal renal function
- History of prolonged QTcF > 450 msec for males or QTcF > 470 msec for females
- History of solid organ or hematological transplantation
- Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30 days of screening
- Women who are pregnant or lactating, or have plans to become pregnant during the study or within 1 month following the last dose
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo - VX-561 100 mg VX-561 - VX-561 150 mg VX-561 - Ivacaftor IVA - VX-561 20 mg VX-561 -
- Primary Outcome Measures
Name Time Method Change From Baseline in Sweat Chloride at Day 28 From baseline at Day 28
- Secondary Outcome Measures
Name Time Method Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28 From baseline at Day 28 Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 From baseline at Day 28
Trial Locations
- Locations (14)
University of Southern California
🇺🇸Los Angeles, California, United States
Stanford Hospital
🇺🇸Palo Alto, California, United States
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
Washington University
🇺🇸Saint Louis, Missouri, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
New York Medical College
🇺🇸Valhalla, New York, United States
Atlantic Health
🇺🇸Morristown, New Jersey, United States
Children's National Health
🇺🇸Washington, District of Columbia, United States
University of Massachusetts
🇺🇸Worcester, Massachusetts, United States