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Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02971839
Recruitment Status : Terminated (Decision by Sponsor.)
First Posted : November 23, 2016
Results First Posted : August 26, 2020
Last Update Posted : August 26, 2020
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-561 Drug: Placebo Drug: IVA Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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.
Actual Study Start Date : December 2016
Actual Primary Completion Date : August 2017
Actual Study Completion Date : August 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: VX-561 20 mg Drug: VX-561
Other Name: CTP-656

Experimental: VX-561 100 mg Drug: VX-561
Other Name: CTP-656

Experimental: VX-561 150 mg Drug: VX-561
Other Name: CTP-656

Active Comparator: Ivacaftor Drug: IVA
Other Names:
  • ivacaftor; VX-770
  • Kalydeco

Placebo Comparator: Placebo Drug: Placebo



Primary Outcome Measures :
  1. Change From Baseline in Sweat Chloride at Day 28 [ Time Frame: From baseline at Day 28 ]

Secondary Outcome Measures :
  1. Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28 [ Time Frame: From baseline at Day 28 ]
  2. Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28 [ Time Frame: From baseline at Day 28 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02971839


Locations
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United States, California
University of Southern California
Los Angeles, California, United States
Stanford Hospital
Palo Alto, California, United States
United States, District of Columbia
Children's National Health
Washington, District of Columbia, United States
United States, Florida
University of Miami
Miami, Florida, United States
United States, Illinois
Rush University
Chicago, Illinois, United States
United States, Indiana
Indiana University
Indianapolis, Indiana, United States
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States
University of Massachusetts
Worcester, Massachusetts, United States
United States, Missouri
Washington University
Saint Louis, Missouri, United States
United States, New Jersey
Atlantic Health
Morristown, New Jersey, United States
United States, New York
New York Medical College
Valhalla, New York, United States
United States, Ohio
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] October 24, 2016
Statistical Analysis Plan  [PDF] September 22, 2017

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02971839    
Other Study ID Numbers: CP656.2001
First Posted: November 23, 2016    Key Record Dates
Results First Posted: August 26, 2020
Last Update Posted: August 26, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action