Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

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

NCT06460506

VX22-445-123

2023-509563-24-00 (Other Identifier)

Details and patient eligibility

About

The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).

Enrollment: 50 patients

Sex: All

Ages: 12+ months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Completed study drug treatment in the parent study VX22-445-122 Part B (NCT05882357) OR had study drug interruption(s) in the parent study but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the Treatment Period of the parent study

Key Exclusion Criteria:

• History of drug intolerance in the parent study that would pose an additional risk to the participant
• Current participation in an investigational drug trial other than 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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50 participants in 1 patient group

ELX/TEZ/IVA

Experimental group

Description:

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Treatment:

Drug: IVA

Drug: ELX/TEZ/IVA