A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

December 31, 2021 updated by: Vertex Pharmaceuticals Incorporated

A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
77

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

  • Australia
      • Chermside, Australia
        • The Prince Charles Hospital
      • Melbourne, VIC, Australia
        • Alfred Hospital
      • South Brisbane, Australia
        • Mater Adult Hospital
      • Westmead, Australia
        • Westmead Hospital
  • Belgium
      • Gent, Belgium
        • Universitair Ziekenhuis Gent
  • Germany
      • Erlangen, Germany
        • Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
      • Essen, Germany
        • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
      • Halle, Germany
        • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
      • Muenchen, Germany
        • Pneumologisches Studienzentrum Muenchen-West
      • Würzburg, Germany
        • University Hospital Wuerzburg
  • Ireland
      • Dublin, Ireland
        • St. Vincent's University Hospital
      • Dublin 12, Ireland
        • Cork University Hospital
      • Dublin 9, Ireland
        • Beaumont Hospital
      • Limerick, Ireland
        • University Hospital Limerick (Adults)
  • Netherlands
      • Nijmegen, Netherlands
        • UMC St. Radboud
  • United Kingdom
      • Glasgow, United Kingdom
        • Clinical Research Facility, Queen Elizabeth University Hospital
      • Leeds, United Kingdom
        • St. James University Hospital
      • Liverpool, United Kingdom
        • Liverpool Heart and Chest Hospital
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama at Birmingham
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Banner University of Arizona Medical Center
    • California
      • Long Beach, California, United States, 90806
        • Miller Children's Hospital / Long Beach Memorial
      • San Francisco, California, United States, 94143
        • UCSF Gateway Medical Center
    • Colorado
      • Denver, Colorado, United States, 80206
        • National Jewish Health
    • Florida
      • Miami, Florida, United States, 33136
        • University of Miami Miller School of Medicine
      • Orlando, Florida, United States, 32803
        • Central Florida Pulmonary Group, PA
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • University of Kentucky
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Boston Children's Hospital
      • Boston, Massachusetts, United States, 02114
        • Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-5212
        • Michigan Medicine
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • University of Minnesota
    • Missouri
      • Saint Louis, Missouri, United States, 63104
        • Cardinal Glennon Children's Hospital
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine / St. Louis Children's Hospital
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth Hitchcock Medical Center
    • New York
      • New York, New York, United States, 10032
        • Columbia University Medical Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • University of North Carolina Hospitals
    • Ohio
      • Cincinnati, Ohio, United States, 45220
        • UC Health Holmes
      • Toledo, Ohio, United States, 43606
        • ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • University of Oklahoma Health Sciences Center
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
    • Texas
      • Austin, Texas, United States, 78723
        • Dell Children's Medical Group
      • Dallas, Texas, United States, 75390
        • The University of Texas Southwestern Medical Center
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah / Primary Children's Medical Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • University of Wisconsin Hospital and Clinics

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
  • On ivacaftor therapy
  • FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • History of clinically significant cirrhosis with or without portal hypertension
  • History of solid organ or hematological transplantation
  • Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Active Comparator: Ivacaftor
Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
Drug: IVA
150-mg film-coated tablet for oral administration.
Other Names:
  • VX-770
  • Ivacaftor
Drug: Placebo
Placebo matched to IVA.
Drug: Placebo
Placebos matched to VX-561.
Experimental: VX-561: 25 mg
Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.
Drug: Placebo
Placebo matched to IVA.
Drug: Placebo
Placebos matched to VX-561.
Drug: VX-561
VX-561 tablets for oral administration.
Other Names:
  • CTP-656
  • Deutivacaftor (D-IVA)
Experimental: VX-561: 50 mg
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
Drug: Placebo
Placebo matched to IVA.
Drug: Placebo
Placebos matched to VX-561.
Drug: VX-561
VX-561 tablets for oral administration.
Other Names:
  • CTP-656
  • Deutivacaftor (D-IVA)
Experimental: VX-561: 150 mg
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
Drug: Placebo
Placebo matched to IVA.
Drug: Placebo
Placebos matched to VX-561.
Drug: VX-561
VX-561 tablets for oral administration.
Other Names:
  • CTP-656
  • Deutivacaftor (D-IVA)
Experimental: VX-561: 250 mg
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
Drug: Placebo
Placebo matched to IVA.
Drug: Placebo
Placebos matched to VX-561.
Drug: VX-561
VX-561 tablets for oral administration.
Other Names:
  • CTP-656
  • Deutivacaftor (D-IVA)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Time Frame: From Baseline at Week 12
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
From Baseline at Week 12

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Absolute Change in Sweat Chloride (SwCl)
Time Frame: From Baseline at Week 12
Sweat samples were collected using an approved collection device.
From Baseline at Week 12
Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)
Time Frame: At Week 4
At Week 4
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Baseline up to Week 16
Baseline up to Week 16

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 17, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 20, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 20, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 9, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 9, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 11, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 25, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 31, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • VX18-561-101
  • 2018-003970-28 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

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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