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

Vertex Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: ELX/TEZ/IVA; Drug: IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT05331183

VX21-445-125

2021-005914-33 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 297 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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297 participants in 1 patient group

ELX/TEZ/IVA

Experimental group

Description:

Part A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks. Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.

Treatment:

Drug: IVA

Drug: ELX/TEZ/IVA