A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

Vertex Pharmaceuticals Incorporated67 enrolled

Overview

This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Cystic Fibrosis

Interventions

TEZ/IVADRUG

Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.

IVADRUG

Participants weighing \<40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.

PlaceboDRUG

Placebo matched to TEZ/IVA FDC

Eligibility

Sex: ALLMin age: 6 YearsMax age: 11 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol). * Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height. * Participants with a screening LCI2.5 result ≥7.5. * Participants who are able to swallow tablets. Key Exclusion Criteria: * Clinically significant cirrhosis with or without portal hypertension. * Colonization 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 (27)

Hunter Medical Research Institute (HMRI)

New Lambton Heights, Australia

Outcomes

Primary Outcomes

Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8

LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

Time frame: From baseline through Week 8

Secondary Outcomes

Absolute Change in Sweat Chloride At Week 8

Sweat samples were collected using an approved collection device.

Time frame: From baseline at Week 8

Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score 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: From baseline through Week 8

Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit

Time frame: From first dose of study drug up to safety follow-up visit (up to Week 12)

Data from ClinicalTrials.gov

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