Study Stopped
Decision by Sponsor.
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
A Phase 2, Randomized, Parallel-Group, Double-Blind, Placebo Controlled Study to Evaluate the Safety and Efficacy of CTP-656 With an Open-Label Active Comparator in Patients With Cystic Fibrosis With CFTR Gating Mutations.
1 other identifier
interventional
11
1 country
14
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Dec 2016
Shorter than P25 for phase_2
14 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 21, 2016
CompletedFirst Posted
Study publicly available on registry
November 23, 2016
CompletedStudy Start
First participant enrolled
December 1, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2017
CompletedResults Posted
Study results publicly available
August 26, 2020
CompletedAugust 26, 2020
August 1, 2020
8 months
November 21, 2016
August 11, 2020
August 11, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Change From Baseline in Sweat Chloride at Day 28
From baseline at Day 28
Secondary Outcomes (2)
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
From baseline at Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28
From baseline at Day 28
Study Arms (5)
VX-561 20 mg
EXPERIMENTALVX-561 100 mg
EXPERIMENTALVX-561 150 mg
EXPERIMENTALIvacaftor
ACTIVE COMPARATORPlacebo
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (14)
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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 21, 2016
First Posted
November 23, 2016
Study Start
December 1, 2016
Primary Completion
August 1, 2017
Study Completion
August 1, 2017
Last Updated
August 26, 2020
Results First Posted
August 26, 2020
Record last verified: 2020-08
Data Sharing
- IPD Sharing
- Will not share