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A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

NCT ID: NCT03460990

Last Updated: 2019-10-17

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

116 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-05-01

Study Completion Date

2018-10-08

Brief Summary

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This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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TEZ/IVA

Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the triple combination (TC) treatment period.

Group Type ACTIVE_COMPARATOR

TEZ/IVA

Intervention Type DRUG

Participants received TEZ/IVA orally once daily in the morning.

IVA

Intervention Type DRUG

Participants received IVA orally once daily in the evening.

Placebo

Intervention Type DRUG

Participants received placebo matched to VX-659/TEZ/IVA orally once daily in the morning.

VX-659/TEZ/IVA TC

Following a run-in period of 4 weeks with TEZ/IVA, participants received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the TC treatment period.

Group Type EXPERIMENTAL

VX-659/TEZ/IVA

Intervention Type DRUG

Participants received VX-659/TEZ/IVA orally once daily in the morning.

IVA

Intervention Type DRUG

Participants received IVA orally once daily in the evening.

Placebo

Intervention Type DRUG

Participants received placebo matched TEZ/IVA orally once daily in the morning.

Interventions

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VX-659/TEZ/IVA

Participants received VX-659/TEZ/IVA orally once daily in the morning.

Intervention Type DRUG

TEZ/IVA

Participants received TEZ/IVA orally once daily in the morning.

Intervention Type DRUG

IVA

Participants received IVA orally once daily in the evening.

Intervention Type DRUG

Placebo

Participants received placebo matched TEZ/IVA orally once daily in the morning.

Intervention Type DRUG

Placebo

Participants received placebo matched to VX-659/TEZ/IVA orally once daily in the morning.

Intervention Type DRUG

Other Intervention Names

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

Eligibility Criteria

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Inclusion Criteria

* Homozygous for the F508del mutation (F/F)
* Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Exclusion Criteria

* Clinically significant cirrhosis with or without portal hypertension
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Solid organ or hematological transplantation
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Yale New Haven Medical Center

New Haven, Connecticut, United States

Site Status

University of Miami/Miller School of Medicine

Miami, Florida, United States

Site Status

St. Luke's CF Center of Idaho

Boise, Idaho, United States

Site Status

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

Niles, Illinois, United States

Site Status

Indiana Clinical Research Center, IU Health University Hospital

Indianapolis, Indiana, United States

Site Status

The University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

The Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Helen DeVos Children's Hospital CF Center

Grand Rapids, Michigan, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Washington University School of Medicine / St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status

Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

Northwell Health, Long Island Jewish Medical Center

New Hyde Park, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Respiratory Diseases of Children and Adolescents

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

Sanford Research/ USD

Sioux Falls, South Dakota, United States

Site Status

University of Tennessee Medical Center-Adult Cystic Fibrosis Clinic

Knoxville, Tennessee, United States

Site Status

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

Memphis, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

University of Utah/ Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

The Alfred Hospital

Melbourne, Victoria, Australia

Site Status

Royal Adelaide Hospital

Adelaide, , Australia

Site Status

Prince Charles Hospital

Chermside, , Australia

Site Status

Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital

Nedlands, , Australia

Site Status

John Hunter Hospital & Hunter Medical Research Institute

New Lambton Heights, , Australia

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Pneumologische Praxis Pasing

München, , Germany

Site Status

Cork University Hospital

Dublin, , Ireland

Site Status

Our Lady's Children's Hospital

Dublin, , Ireland

Site Status

St. Vincent's University Hospital

Dublin, , Ireland

Site Status

Hospital Universitari Vall d Hebron

Barcelona, , Spain

Site Status

Hospital Universitari Vall d'Hebron Servicio de Broncoscopia

Barcelona, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Papworth Hospital NHS Foundation Trust, Papworth Everard

Cambridge, , United Kingdom

Site Status

Liverpool Heart and Chest Hospital

Liverpool, , United Kingdom

Site Status

Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

London, , United Kingdom

Site Status

Wythenshawe Hospital

Manchester, , United Kingdom

Site Status

University Hospital Llandough

Penarth, , United Kingdom

Site Status

Countries

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United States Australia Germany Ireland Spain United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2017-004133-82

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX17-659-103

Identifier Type: -

Identifier Source: org_study_id

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