A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor

Vertex Pharmaceuticals Incorporated156 enrolled

Overview

This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

156

Conditions

Cystic Fibrosis

Interventions

IvacaftorDRUG

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive * FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height during screening * Stable CF disease as judged by the investigator. Exclusion Criteria: * History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject. * Pregnant and nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Week -4 Visits). * Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements

Locations (68)

Unnamed facility

Birmingham, Alabama, United States

Unnamed facility

Oakland, California, United States

Unnamed facility

San Diego, California, United States

Unnamed facility

Aurora, Colorado, United States

Unnamed facility

Washington D.C., District of Columbia, United States

Unnamed facility

Outcomes

Primary Outcomes

Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time frame: Baseline, Through Week 8

Secondary Outcomes

Relative Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Time frame: Baseline, Through Week 8

Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline Through Week 8

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.

Time frame: Baseline, Through Week 8

Absolute Change From Baseline in Sweat Chloride Through Week 8

Sweat samples were collected using an approved collection device.

Time frame: Baseline, Through Week 8

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Baseline up to Week 16

Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)

Time frame: Predose on Week -2 for Run-in period; Pre-dose on Week 2 for Active comparator period