Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Phase 3Active Not RecruitingNCT05331183

Vertex Pharmaceuticals Incorporated297 enrolled

Overview

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

297

Conditions

Cystic Fibrosis

Interventions

ELX/TEZ/IVADRUG

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

IVADRUG

Tablets for oral administration.

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: Part A: Completed study drug treatment in parent study or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A Key Exclusion Criteria: History of study drug intolerance in the parent study Other protocol defined Inclusion/Exclusion criteria may apply.

Locations (81)

Outcomes

Primary Outcomes

Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time frame: Day 1 up to Week 196