A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy

Vertex Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: VX-121/TEZ/D-IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT05444257

VX20-121-104

2024-514173-22-00 (Other Identifier)

2021-000713-17 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.

Enrollment: 822 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Part A: Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but did not permanently discontinue study drug, and 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 did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part A

Key Exclusion Criteria:

• History of drug intolerance in a parent study
• Pregnant or breast-feeding females at the time of enrollment in Part A

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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822 participants in 1 patient group

VX-121/TEZ/D-IVA

Experimental group

Description:

Part A: Participants will receive VX-121/TEZ/D-IVA once daily for 96 weeks. Part B: Participants will receive VX-121/TEZ/D-IVA once daily for an additional 48 weeks.

Treatment:

Drug: VX-121/TEZ/D-IVA