A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation

Vertex Pharmaceuticals Incorporated130 enrolled

Overview

This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

130

Conditions

Cystic Fibrosis

Interventions

TEZ/IVADRUG

Fixed-dose combination tablet for oral administration.

IVADRUG

Tablet for oral administration.

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115 * Eligible CFTR Mutation Exclusion Criteria: * Pregnant and nursing females * History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator * Ongoing participation in another study with investigational drug Other protocol defined Inclusion/Exclusion criteria may apply.

Locations (55)

Outcomes

Primary Outcomes

Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Time frame: Day 1 up to Week 100

Secondary Outcomes

Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group)

The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.

Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS

Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

Part A: Absolute Change in Sweat Chloride (SwCl) for 115/116 FAS (TEZ/IVA Group)

Sweat samples were collected using an approved collection device.

Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

Part A: Absolute Change in SwCl for 113B/116 FAS

Sweat samples were collected using an approved collection device.

Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

Data from ClinicalTrials.gov

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University of Alabama at Birmingham

Birmingham, Alabama, United States

Providence Alaska Medical Center

Anchorage, Alaska, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Children's Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours/ Alfred I. duPont Hospital for Children

Wilmington, Delaware, United States

Johns Hopkins All Children's Hospital Outpatient Care Center

St. Petersburg, Florida, United States

Center for Advanced Pediatrics

Atlanta, Georgia, United States

St. Luke's CF Center of Idaho

Boise, Idaho, United States

Riley Hospital for Children Indiana University Health

Indianapolis, Indiana, United States

...and 45 more locations

Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group)

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS

Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group)

BMI was defined as weight in kilograms (kg) divided by squared height in meters (m\^2).

Time frame: From Parent Study 115 Baseline at Week 96 (Study 116)

Part A: Absolute Change in BMI for 113B/116 FAS

BMI was defined as weight in kg divided by m\^2.

Time frame: From Parent Study 113B Baseline at Week 96 (Study 116)

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

Time frame: Day 1 up to Week 192