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

August 11, 2020 updated by: Vertex Pharmaceuticals Incorporated

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.

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

11

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States
        • University of Southern California
      • Palo Alto, California, United States
        • Stanford Hospital
    • District of Columbia
      • Washington, District of Columbia, United States
        • Children's National Health
    • Florida
      • Miami, Florida, United States
        • University of Miami
    • Illinois
      • Chicago, Illinois, United States
        • Rush University
    • Indiana
      • Indianapolis, Indiana, United States
        • Indiana University
    • Massachusetts
      • Boston, Massachusetts, United States
        • Boston Children's Hospital
      • Worcester, Massachusetts, United States
        • University of Massachusetts
    • Missouri
      • Saint Louis, Missouri, United States
        • Washington University
    • New Jersey
      • Morristown, New Jersey, United States
        • Atlantic Health
    • New York
      • Valhalla, New York, United States
        • New York Medical College
    • Ohio
      • Cincinnati, Ohio, United States
        • Cincinnati Children's Hospital
      • Columbus, Ohio, United States
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States
        • Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
Experimental: VX-561 20 mg
Drug: VX-561
Other Names:
  • CTP-656
Experimental: VX-561 100 mg
Drug: VX-561
Other Names:
  • CTP-656
Experimental: VX-561 150 mg
Drug: VX-561
Other Names:
  • CTP-656
Active Comparator: Ivacaftor
Drug: IVA
Other Names:
  • ivacaftor; VX-770
  • Kalydeco

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline in Sweat Chloride at Day 28
Time Frame: From baseline at Day 28
From baseline at Day 28

Secondary Outcome Measures

Outcome Measure
Time Frame
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Time Frame: From baseline at Day 28
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
From baseline at Day 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 1, 2016

Primary Completion (Actual)

August 1, 2017

Study Completion (Actual)

August 1, 2017

Study Registration Dates

First Submitted

November 21, 2016

First Submitted That Met QC Criteria

November 21, 2016

First Posted (Estimate)

November 23, 2016

Study Record Updates

Last Update Posted (Actual)

August 26, 2020

Last Update Submitted That Met QC Criteria

August 11, 2020

Last Verified

August 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CP656.2001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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