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A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

Primary Purpose

Cystic Fibrosis

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

VX-659/TEZ/IVA

IVA

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

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

Inclusion Criteria:

Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.

Exclusion Criteria:

History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
Current participation in an investigational drug trial (other than a parent study)

Other protocol defined Inclusion/Exclusion criteria may apply.

Sites / Locations

University of Alabama at Birmingham
Stanford University
Children's Hospital Colorado
Hartford Hospital
Yale New Haven Medical Center
University of Miami Miller School of Medicine
Nicklaus Children's Hospital
Orlando Health, Inc.- Arnold Palmer Hospital for Children (APH)
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Luke's CF Center of Idaho
Cystic Fibrosis Center of Chicago
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
Indiana Clinical Research Center, IU Health University Hospital
The University of Iowa Hospitals and Clinics
University of Kentucky
Kosair Charities Pediatric Clinical Research Unit
Johns Hopkins Hospital
Boston Children's Hospital
UMass Memorial Medical Center
Michigan Medicine
Spectrum Health Medical Group Adult Cystic Fibrosis Care Center
University of Minnesota
University of Mississippi Medical Center
The Children's Mercy Hospital
Washington University School of Medicine/ St. Louis Children's Hospital
Dartmouth Hitchcock, Manchester
Rutgers-Robert Wood Johnson Medical School
Albany Medical College
CF Therapeutics Development Center of Western New York
Northwell Health, Long Island Jewish Medical Center
Columbia University Medical Center
SUNY Upstate Medical University
Clinical Research of Charlotte
Duke University Medical Center
Cincinnati Children's Hospital
Santiago Reyes, M.D.
Oregon Health & Science University
Drexel University College of Medicine / Drexel Adult Cystic Fibrosis Center
Children's Hospital of Pittsburgh of UPMC
Sanford Children's Specialty Clinic
University of Tennessee Medical Center- Adult Cystic Fibrosis Clinic
Children's Foundation Research Center / Le Bonheur Children's Hospital
Vanderbilt University Medical Center
Cook Children's Medical Center
Texas Children's Hospital
University of Utah/ Primary Children's Medical Center
Seattle Children's Hospital
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
Royal Adelaide Hospital
The Prince Charles Hospital
Alfred Hospital
Institute for Respiratory Health, Sir Charles Gairdner Hospital
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
Telethon Kids Institute
Sydney Children's Hospital
Lady Cilento Children's Hospital
Stollery Children's Hospital
Queen Elizabeth II Health Sciences Center
St. Michael's Hospital
Juliane Marie Center, Rigshospitalet
Charite Paediatric Pulmonology Department
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Clinic of J.W. Goethe University
Medizinische Hochschule Hannover
Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
Universitaetsklinkum Koeln, CF-Studienzentrum
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
Pneumologische Praxis Pasing
Klinikum Innenstadt, University of Munich
Cork University Hospital
Beaumont Hospital
Our Lady's Children's Hospital
St. Vincent's University Hospital
Temple Street Children's University Hospital
University Hospital Galway
University Hospital Limerick
Lady Davis Carmel Medical Center
Rambam Health Care Campus, Liver Unit
Pediatrics Hadassah Medical Center
Schneider Children's Medical Center
Sheba Medical Center
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
Hospital Universitari Vall d Hebron
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Hospital Universitario 12 de Octubre
Hospital Universitario Infantil La Paz
Parc Tauli Sabadell Hospital Universitari
Hospital Universitario Virgen del Rocio
Hospital Universitario y Politecnico La Fe
Lindenhofspital - Quartier Bleu
Kinderspital Zuerich
Papworth Hospital NHS Foundation Trust, Papworth Everard
Clinical Research Facility, Queen Elizabeth University Hospital
St. James University Hospital
Liverpool Head and Chest Hospital
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
Wythenshaw e Hospital
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Wolfson Cystic Fibrosis Unit, City Campus
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VX-659/TEZ/IVA TC

Arm Description

Participants from parent studies VX17-659-102 (NCT03447249) or VX17-659-103 (NCT03460990) were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study VX17-659-105.

Outcomes

Primary Outcome Measures

Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in ppFEV1 for Participants From the Parent Study 659-103

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102

Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in SwCl for Participants From the Parent Study 659-103

Sweat samples were collected using an approved collection device. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102

PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

Number of PEx for Participants From the Parent Study 659-103

PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported for overall participants from the parent study 659-103, that is combined for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

Number of Participants With at Least One PEx for Participants From the Parent Study 659-102

PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the Kaplan-Meier (KM) method. However, because way less than 50% of participants had events, median time-to-first event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported separately for those in Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-102 or/and VX-659/TEZ/IVA in the current study 659-105). Baseline was defined as the parent study baseline except for Placebo - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

Number of Participants With at Least One PEx for Participants From the Parent Study 659-103

PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the KM method. However, because way less than 50% of participants had events, median time-to-first-event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported for all participants from the parent study 659-103, that is combined for those in the TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105). Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102

BMI was defined as weight in kilograms (kg) divided by squared height in meters (m^2). The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in BMI for Participants From the Parent Study 659-103

BMI was defined as weight in kg divided by squared height in meters (m^2). The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in BMI Z-score for Participants From the Parent Study 659-102 (Participants <=20 Years Old at Parent Study Baseline)

BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in BMI Z-score for Participants From The Parent Study 659-103 (Participants <=20 Years Old at Parent Study Baseline)

BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in Body Weight for Participants From the Parent Study 659-102

The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in Body Weight for Participants From the Parent Study 659-103

The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for Participants From the Parent Study 659-102

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. The analysis was planned to be reported separately for Placebo - VX-659/TEZ/IVA category (participants who received placebo in the parent study 659-102 and VX-659/TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-102 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Absolute Change in CFQ-R Respiratory Domain Score for Participants From the Parent Study 659-103

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Full Information

NCT ID

NCT03447262

First Posted

February 21, 2018

Last Updated

December 31, 2021

Sponsor

Vertex Pharmaceuticals Incorporated

1. Study Identification

Unique Protocol Identification Number

NCT03447262

Brief Title

A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

Official Title

A Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation

Study Type

Interventional

2. Study Status

Record Verification Date

December 2021

Overall Recruitment Status

Terminated

Why Stopped

At Sponsor's Discretion

Study Start Date

July 13, 2018 (Actual)

Primary Completion Date

September 9, 2020 (Actual)

Study Completion Date

September 9, 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

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.

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 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

484 (Actual)

8. Arms, Groups, and Interventions

Arm Title

VX-659/TEZ/IVA TC

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

VX-659/TEZ/IVA

Other Intervention Name(s)

VX-659/VX-661/VX-770, VX-659/tezacaftor/ivacaftor

Intervention Description

Fixed-dose combination tablets for oral administration qd in the morning.

Intervention Type

Drug

Intervention Name(s)

IVA

Other Intervention Name(s)

VX-770, ivacaftor

Intervention Description

IVA tablet qd in the evening.

Primary Outcome Measure Information:

Title

Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time Frame

From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100)

Secondary Outcome Measure Information:

Title

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102

Description

Time Frame

From Baseline at Week 72 (Study 659-105)

Title

Absolute Change in ppFEV1 for Participants From the Parent Study 659-103

Description

Time Frame

From Baseline at Week 72 (Study 659-105)

Title

Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102

Description

Time Frame

From Baseline at Week 24 (Study 659-105)

Title

Absolute Change in SwCl for Participants From the Parent Study 659-103

Description

Time Frame

From Baseline at Week 24 (Study 659-105)

Title

Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102

Description

Time Frame

From Baseline up to Week 96 (Study 659-105)

Title

Number of PEx for Participants From the Parent Study 659-103

Description

PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The analysis was planned to be reported for overall participants from the parent study 659-103, that is combined for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

Time Frame

From Baseline up to Week 96 (Study 659-105)

Title

Number of Participants With at Least One PEx for Participants From the Parent Study 659-102

Description

Time Frame

From Baseline up to Week 96 (Study 659-105)

Title

Number of Participants With at Least One PEx for Participants From the Parent Study 659-103

Description

PEx was defined as treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for at least 4 sinopulmonary signs/symptoms. The time-to-first-PEx data were planned to be estimated using the KM method. However, because way less than 50% of participants had events, median time-to-first-event data were not estimable. Instead, the number of participants with at least one PEx event was assessed and reported for all participants from the parent study 659-103, that is combined for those in the TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and the VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in the parent study 659-103 or/and in the current study 659-105). Baseline was defined as the parent study baseline except for TEZ/IVA - VX-659/TEZ/IVA category, for which the baseline was defined as study 659-105 baseline.

Time Frame

From Baseline up to Week 96 (Study 659-105)

Title

Absolute Change in Body Mass Index (BMI) for Participants From the Parent Study 659-102

Description

Time Frame

From Baseline at Week 72 (Study 659-105)

Title

Absolute Change in BMI for Participants From the Parent Study 659-103

Description

Time Frame

From Baseline at Week 72 (Study 659-105)

Title

Absolute Change in BMI Z-score for Participants From the Parent Study 659-102 (Participants <=20 Years Old at Parent Study Baseline)

Description

Time Frame

From Baseline at Week 60 (Study 659-105)

Title

Absolute Change in BMI Z-score for Participants From The Parent Study 659-103 (Participants <=20 Years Old at Parent Study Baseline)

Description

BMI was defined as weight in kg divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Time Frame

From Baseline at Week 60 (Study 659-105)

Title

Absolute Change in Body Weight for Participants From the Parent Study 659-102

Description

Time Frame

From Baseline at Week 72 (Study 659-105)

Title

Absolute Change in Body Weight for Participants From the Parent Study 659-103

Description

Time Frame

From Baseline at Week 72 (Study 659-105)

Title

Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for Participants From the Parent Study 659-102

Description

Time Frame

From Baseline at Week 72 (Study 659-105)

Title

Absolute Change in CFQ-R Respiratory Domain Score for Participants From the Parent Study 659-103

Description

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. The analysis was planned to be reported separately for TEZ/IVA - VX-659/TEZ/IVA category (participants who received TEZ/IVA in the parent study 659-103 and VX-659-TEZ/IVA in the current study 659-105) and VX-659/TEZ/IVA - VX-659/TEZ/IVA category (participants who received VX-659/TEZ/IVA in both the parent study 659-103 and in the current study 659-105) as pre-specified in analysis plan. Baseline was defined as the parent study baseline.

Time Frame

From Baseline at Week 72 (Study 659-105)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study. Exclusion Criteria: History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator. Current participation in an investigational drug trial (other than a parent study) 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

Stanford University

City

Palo Alto

State/Province

California

ZIP/Postal Code

94304

Country

United States

Facility Name

Children's Hospital Colorado

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

Hartford Hospital

City

Hartford

State/Province

Connecticut

ZIP/Postal Code

06102

Country

United States

Facility Name

Yale New Haven Medical Center

City

New Haven

State/Province

Connecticut

ZIP/Postal Code

06511

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

Nicklaus Children's Hospital

City

Miami

State/Province

Florida

ZIP/Postal Code

33155

Country

United States

Facility Name

Orlando Health, Inc.- Arnold Palmer Hospital for Children (APH)

City

Orlando

State/Province

Florida

ZIP/Postal Code

32806

Country

United States

Facility Name

Johns Hopkins All Children's Hospital Outpatient Care Center

City

Saint Petersburg

State/Province

Florida

ZIP/Postal Code

33701

Country

United States

Facility Name

St. Luke's CF Center of Idaho

City

Boise

State/Province

Idaho

ZIP/Postal Code

83712

Country

United States

Facility Name

Cystic Fibrosis Center of Chicago

City

Glenview

State/Province

Illinois

ZIP/Postal Code

60025

Country

United States

Facility Name

Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants

City

Niles

State/Province

Illinois

ZIP/Postal Code

60714

Country

United States

Facility Name

Indiana Clinical Research Center, IU Health University Hospital

City

Indianapolis

State/Province

Indiana

ZIP/Postal Code

46202

Country

United States

Facility Name

The University of Iowa Hospitals and Clinics

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

University of Kentucky

City

Lexington

State/Province

Kentucky

ZIP/Postal Code

40536

Country

United States

Facility Name

Kosair Charities Pediatric Clinical Research Unit

City

Louisville

State/Province

Kentucky

ZIP/Postal Code

40202

Country

United States

Facility Name

Johns Hopkins Hospital

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21287

Country

United States

Facility Name

Boston Children's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

UMass Memorial Medical Center

City

Worcester

State/Province

Massachusetts

ZIP/Postal Code

01655

Country

United States

Facility Name

Michigan Medicine

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48109-5212

Country

United States

Facility Name

Spectrum Health Medical Group Adult Cystic Fibrosis Care Center

City

Grand Rapids

State/Province

Michigan

ZIP/Postal Code

49546

Country

United States

Facility Name

University of Minnesota

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

Facility Name

University of Mississippi Medical Center

City

Jackson

State/Province

Mississippi

ZIP/Postal Code

39216

Country

United States

Facility Name

The Children's Mercy Hospital

City

Kansas City

State/Province

Missouri

ZIP/Postal Code

64108

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, Manchester

City

Manchester

State/Province

New Hampshire

ZIP/Postal Code

03104

Country

United States

Facility Name

Rutgers-Robert Wood Johnson Medical School

City

New Brunswick

State/Province

New Jersey

ZIP/Postal Code

08901

Country

United States

Facility Name

Albany Medical College

City

Albany

State/Province

New York

ZIP/Postal Code

12208

Country

United States

Facility Name

CF Therapeutics Development Center of Western New York

City

Buffalo

State/Province

New York

ZIP/Postal Code

14203

Country

United States

Facility Name

Northwell Health, Long Island Jewish Medical Center

City

New Hyde Park

State/Province

New York

ZIP/Postal Code

11040

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

SUNY Upstate Medical University

City

Syracuse

State/Province

New York

ZIP/Postal Code

13210

Country

United States

Facility Name

Clinical Research of Charlotte

City

Charlotte

State/Province

North Carolina

ZIP/Postal Code

28277

Country

United States

Facility Name

Duke University Medical Center

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27710

Country

United States

Facility Name

Cincinnati Children's Hospital

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

Facility Name

Santiago Reyes, M.D.

City

Oklahoma City

State/Province

Oklahoma

ZIP/Postal Code

73112

Country

United States

Facility Name

Oregon Health & Science University

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

Drexel University College of Medicine / Drexel Adult Cystic Fibrosis Center

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19107

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

Sanford Children's Specialty Clinic

City

Sioux Falls

State/Province

South Dakota

ZIP/Postal Code

57105

Country

United States

Facility Name

University of Tennessee Medical Center- Adult Cystic Fibrosis Clinic

City

Knoxville

State/Province

Tennessee

ZIP/Postal Code

37920

Country

United States

Facility Name

Children's Foundation Research Center / Le Bonheur Children's Hospital

City

Memphis

State/Province

Tennessee

ZIP/Postal Code

38103

Country

United States

Facility Name

Vanderbilt University Medical Center

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37232

Country

United States

Facility Name

Cook Children's Medical Center

City

Fort Worth

State/Province

Texas

ZIP/Postal Code

76104

Country

United States

Facility Name

Texas Children's Hospital

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

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

Seattle Children's Hospital

City

Seattle

State/Province

Washington

ZIP/Postal Code

98105

Country

United States

Facility Name

Providence Pediatric Pulmonary & Cystic Fibrosis Clinic

City

Spokane

State/Province

Washington

ZIP/Postal Code

99204

Country

United States

Facility Name

Royal Adelaide Hospital

City

Adelaide

Country

Australia

Facility Name

The Prince Charles Hospital

City

Chermside

Country

Australia

Facility Name

Alfred Hospital

City

Melbourne

Country

Australia

Facility Name

Institute for Respiratory Health, Sir Charles Gairdner Hospital

City

Nedlands

Country

Australia

Facility Name

John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital

City

New Lambton

Country

Australia

Facility Name

Telethon Kids Institute

City

Perth

Country

Australia

Facility Name

Sydney Children's Hospital

City

Randwick

Country

Australia

Facility Name

Lady Cilento Children's Hospital

City

South Brisbane

Country

Australia

Facility Name

Stollery Children's Hospital

City

Edmonton

Country

Canada

Facility Name

Queen Elizabeth II Health Sciences Center

City

Halifax

Country

Canada

Facility Name

St. Michael's Hospital

City

Toronto

Country

Canada

Facility Name

Juliane Marie Center, Rigshospitalet

City

Copenhagen

Country

Denmark

Facility Name

Charite Paediatric Pulmonology Department

City

Berlin

Country

Germany

Facility Name

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

City

Essen

Country

Germany

Facility Name

Clinic of J.W. Goethe University

City

Frankfurt

Country

Germany

Facility Name

Medizinische Hochschule Hannover

City

Hannover

Country

Germany

Facility Name

Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin

City

Jena

Country

Germany

Facility Name

Universitaetsklinkum Koeln, CF-Studienzentrum

City

Koeln

Country

Germany

Facility Name

Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin

City

Lubeck

Country

Germany

Facility Name

Pneumologische Praxis Pasing

City

Muenchen

Country

Germany

Facility Name

Klinikum Innenstadt, University of Munich

City

München

Country

Germany

Facility Name

Cork University Hospital

City

Cork

Country

Ireland

Facility Name

Beaumont Hospital

City

Dublin

Country

Ireland

Facility Name

Our Lady's Children's Hospital

City

Dublin

Country

Ireland

Facility Name

St. Vincent's University Hospital

City

Dublin

Country

Ireland

Facility Name

Temple Street Children's University Hospital

City

Dublin

Country

Ireland

Facility Name

University Hospital Galway

City

Galway

Country

Ireland

Facility Name

University Hospital Limerick

City

Limerick

Country

Ireland

Facility Name

Lady Davis Carmel Medical Center

City

Haifa

Country

Israel

Facility Name

Rambam Health Care Campus, Liver Unit

City

Haifa

Country

Israel

Facility Name

Pediatrics Hadassah Medical Center

City

Jerusalem

Country

Israel

Facility Name

Schneider Children's Medical Center

City

Petah Tikva

Country

Israel

Facility Name

Sheba Medical Center

City

Tel HaShomer

Country

Israel

Facility Name

Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY

City

Łomianki

Country

Poland

Facility Name

Hospital Universitari Vall d Hebron

City

Barcelona

Country

Spain

Facility Name

Hospital Universitari Vall d´Hebron Servicio de Broncoscopia

City

Barcelona

Country

Spain

Facility Name

Hospital Universitario 12 de Octubre

City

Madrid

Country

Spain

Facility Name

Hospital Universitario Infantil La Paz

City

Madrid

Country

Spain

Facility Name

Parc Tauli Sabadell Hospital Universitari

City

Sabadell

Country

Spain

Facility Name

Hospital Universitario Virgen del Rocio

City

Sevilla

Country

Spain

Facility Name

Hospital Universitario y Politecnico La Fe

City

Valencia

Country

Spain

Facility Name

Lindenhofspital - Quartier Bleu

City

Bern

Country

Switzerland

Facility Name

Kinderspital Zuerich

City

Zürich

Country

Switzerland

Facility Name

Papworth Hospital NHS Foundation Trust, Papworth Everard

City

Cambridge

Country

United Kingdom

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 Head and Chest Hospital

City

Liverpool

Country

United Kingdom

Facility Name

Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

City

London

Country

United Kingdom

Facility Name

Wythenshaw e Hospital

City

Manchester

Country

United Kingdom

Facility Name

The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary

City

Newcastle Upon Tyne

Country

United Kingdom

Facility Name

Wolfson Cystic Fibrosis Unit, City Campus

City

Nottingham

Country

United Kingdom

Facility Name

All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough

City

Penarth

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

33331662

Citation

Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

Results Reference

derived

Learn more about this trial

A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

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