Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age

Vertex Pharmaceuticals

Status and phase: Active, not recruiting; Phase 3

Conditions: Cystic Fibrosis

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT05882357

VX22-445-122

2023-503230-49-00 (Other Identifier)

Details and patient eligibility

About

This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (<) 24 months of age.

Enrollment: 70 patients

Sex: All

Ages: 12 to 24 months old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation

Key Exclusion Criteria:

History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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70 participants in 2 patient groups

Part A

Experimental group

Description:

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

Treatment:

Drug: ELX/TEZ/IVA

Drug: IVA

Part B

Experimental group

Description:

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.

Treatment:

Drug: ELX/TEZ/IVA

Drug: IVA