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

Primary Purpose

Cystic Fibrosis

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
VX-561
Placebo
IVA
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Southern California
  • Stanford Hospital
  • Children's National Health
  • University of Miami
  • Rush University
  • Indiana University
  • Boston Children's Hospital
  • University of Massachusetts
  • Washington University
  • Atlantic Health
  • New York Medical College
  • Cincinnati Children's Hospital
  • Nationwide Children's Hospital
  • Children's Hospital of Philadelphia

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Active Comparator

Placebo Comparator

Arm Label

VX-561 20 mg

VX-561 100 mg

VX-561 150 mg

Ivacaftor

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Change From Baseline in Sweat Chloride at Day 28

Secondary Outcome Measures

Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28

Full Information

First Posted
November 21, 2016
Last Updated
August 11, 2020
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT02971839
Brief Title
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
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.
Study Type
Interventional

2. Study Status

Record Verification Date
August 2020
Overall Recruitment Status
Terminated
Why Stopped
Decision by Sponsor.
Study Start Date
December 2016 (Actual)
Primary Completion Date
August 2017 (Actual)
Study Completion Date
August 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VX-561 20 mg
Arm Type
Experimental
Arm Title
VX-561 100 mg
Arm Type
Experimental
Arm Title
VX-561 150 mg
Arm Type
Experimental
Arm Title
Ivacaftor
Arm Type
Active Comparator
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
VX-561
Other Intervention Name(s)
CTP-656
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
ivacaftor; VX-770, Kalydeco
Primary Outcome Measure Information:
Title
Change From Baseline in Sweat Chloride at Day 28
Time Frame
From baseline at Day 28
Secondary Outcome Measure Information:
Title
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
Time Frame
From baseline at Day 28
Title
Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28
Time Frame
From baseline at Day 28

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
University of Southern California
City
Los Angeles
State/Province
California
Country
United States
Facility Name
Stanford Hospital
City
Palo Alto
State/Province
California
Country
United States
Facility Name
Children's National Health
City
Washington
State/Province
District of Columbia
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
Country
United States
Facility Name
Rush University
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
Indiana University
City
Indianapolis
State/Province
Indiana
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
University of Massachusetts
City
Worcester
State/Province
Massachusetts
Country
United States
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
Country
United States
Facility Name
Atlantic Health
City
Morristown
State/Province
New Jersey
Country
United States
Facility Name
New York Medical College
City
Valhalla
State/Province
New York
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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