A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Phase 3CompletedNCT04058353

Vertex Pharmaceuticals Incorporated271 enrolled

Overview

This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

271

Conditions

Cystic Fibrosis

Interventions

ELX/TEZ/IVADRUG

FDC tablet for oral administration.

IVADRUG

Mono-tablet for oral administration.

Fixed-dose combination (FDC) tablet for oral administration.

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Subject has a confirmed diagnosis of CF and is heterozygous for F508del and either a gating or residual function mutation (F/G and F/RF genotypes) * Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height Key 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 Other protocol defined Inclusion/Exclusion criteria may apply

Locations (93)

Outcomes

Primary Outcomes

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for ELX/TEZ/IVA Group

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

Time frame: From Baseline Through Week 8

Secondary Outcomes

Absolute Change in Sweat Chloride (SwCl) for ELX/TEZ/IVA Group

Sweat samples were collected using an approved collection device.

Time frame: From Baseline Through Week 8

Absolute Change in ppFEV1 for the ELX/TEZ/IVA Group Compared to the Control Group

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

Time frame: From Baseline Through Week 8

Absolute Change in SwCl for ELX/TEZ/IVA Group Compared to the Control Group

Sweat samples were collected using an approved collection device.

Time frame: From Baseline Through Week 8

Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for ELX/TEZ/IVA Group

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: From Baseline Through Week 8

Absolute Change in CFQ-R Respiratory Domain Score for ELX/TEZ/IVA Group Compared to the Control Group

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.

Data from ClinicalTrials.gov

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NCT04058353 - A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes) | Crick | Crick