Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
NCT ID: NCT02971839
Last Updated: 2020-08-26
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
11 participants
INTERVENTIONAL
2016-12-31
2017-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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VX-561 20 mg
VX-561
VX-561 100 mg
VX-561
VX-561 150 mg
VX-561
Ivacaftor
IVA
Placebo
Placebo
Interventions
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VX-561
Placebo
IVA
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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
18 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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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
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
New York Medical College
Valhalla, New York, 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
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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CP656.2001
Identifier Type: -
Identifier Source: org_study_id
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