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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
VX-561
IVA
Placebo
Placebo
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

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

Sites / Locations

  • University of Alabama at Birmingham
  • Banner University of Arizona Medical Center
  • Miller Children's Hospital / Long Beach Memorial
  • UCSF Gateway Medical Center
  • National Jewish Health
  • University of Miami Miller School of Medicine
  • Central Florida Pulmonary Group, PA
  • Indiana University
  • University of Kansas Medical Center
  • University of Kentucky
  • Johns Hopkins Hospital
  • Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
  • Boston Children's Hospital
  • Michigan Medicine
  • University of Minnesota
  • Cardinal Glennon Children's Hospital
  • Washington University School of Medicine / St. Louis Children's Hospital
  • Dartmouth Hitchcock Medical Center
  • Columbia University Medical Center
  • University of North Carolina Hospitals
  • UC Health Holmes
  • ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
  • University of Oklahoma Health Sciences Center
  • Children's Hospital of Philadelphia
  • Children's Hospital of Pittsburgh of UPMC
  • Medical University of South Carolina
  • Dell Children's Medical Group
  • The University of Texas Southwestern Medical Center
  • University of Utah / Primary Children's Medical Center
  • University of Wisconsin Hospital and Clinics
  • The Prince Charles Hospital
  • Alfred Hospital
  • Mater Adult Hospital
  • Westmead Hospital
  • Universitair Ziekenhuis Gent
  • Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
  • Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
  • Pneumologisches Studienzentrum Muenchen-West
  • University Hospital Wuerzburg
  • Cork University Hospital
  • Beaumont Hospital
  • St. Vincent's University Hospital
  • University Hospital Limerick (Adults)
  • UMC St. Radboud
  • Clinical Research Facility, Queen Elizabeth University Hospital
  • St. James University Hospital
  • Liverpool Heart and Chest Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Active Comparator

Experimental

Experimental

Experimental

Experimental

Arm Label

Ivacaftor

VX-561: 25 mg

VX-561: 50 mg

VX-561: 150 mg

VX-561: 250 mg

Arm Description

Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.

Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.

Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.

Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.

Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.

Outcomes

Primary Outcome Measures

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Secondary Outcome Measures

Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
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)
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Full Information

First Posted
April 9, 2019
Last Updated
December 31, 2021
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT03911713
Brief Title
A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
April 17, 2019 (Actual)
Primary Completion Date
August 20, 2020 (Actual)
Study Completion Date
August 20, 2020 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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

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
77 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Ivacaftor
Arm Type
Active Comparator
Arm Description
Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
Arm Title
VX-561: 25 mg
Arm Type
Experimental
Arm Description
Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.
Arm Title
VX-561: 50 mg
Arm Type
Experimental
Arm Description
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
Arm Title
VX-561: 150 mg
Arm Type
Experimental
Arm Description
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
Arm Title
VX-561: 250 mg
Arm Type
Experimental
Arm Description
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
Intervention Type
Drug
Intervention Name(s)
VX-561
Other Intervention Name(s)
CTP-656, Deutivacaftor (D-IVA)
Intervention Description
VX-561 tablets for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, Ivacaftor
Intervention Description
150-mg film-coated tablet for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to IVA.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebos matched to VX-561.
Primary Outcome Measure Information:
Title
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Description
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time Frame
From Baseline at Week 12
Secondary Outcome Measure Information:
Title
Absolute Change in Sweat Chloride (SwCl)
Description
Sweat samples were collected using an approved collection device.
Time Frame
From Baseline at Week 12
Title
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
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Banner University of Arizona Medical Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Miller Children's Hospital / Long Beach Memorial
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
UCSF Gateway Medical Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Central Florida Pulmonary Group, PA
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Indiana University
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Kentucky
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Michigan Medicine
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-5212
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Cardinal Glennon Children's Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
Washington University School of Medicine / St. Louis Children's Hospital
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Dartmouth Hitchcock Medical Center
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
University of North Carolina Hospitals
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
UC Health Holmes
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45220
Country
United States
Facility Name
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Facility Name
University of Oklahoma Health Sciences Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Dell Children's Medical Group
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
The University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
University of Utah / Primary Children's Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Wisconsin Hospital and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
Alfred Hospital
City
Melbourne, VIC
Country
Australia
Facility Name
Mater Adult Hospital
City
South Brisbane
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
Country
Australia
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
City
Erlangen
Country
Germany
Facility Name
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
City
Essen
Country
Germany
Facility Name
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
City
Halle
Country
Germany
Facility Name
Pneumologisches Studienzentrum Muenchen-West
City
Muenchen
Country
Germany
Facility Name
University Hospital Wuerzburg
City
Würzburg
Country
Germany
Facility Name
Cork University Hospital
City
Dublin 12
Country
Ireland
Facility Name
Beaumont Hospital
City
Dublin 9
Country
Ireland
Facility Name
St. Vincent's University Hospital
City
Dublin
Country
Ireland
Facility Name
University Hospital Limerick (Adults)
City
Limerick
Country
Ireland
Facility Name
UMC St. Radboud
City
Nijmegen
Country
Netherlands
Facility Name
Clinical Research Facility, Queen Elizabeth University Hospital
City
Glasgow
Country
United Kingdom
Facility Name
St. James University Hospital
City
Leeds
Country
United Kingdom
Facility Name
Liverpool Heart and Chest Hospital
City
Liverpool
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing 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

Learn more about this trial

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

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