Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Vertex Pharmaceuticals

Status and phase: Enrolling; Phase 3

Conditions: Cystic Fibrosis

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT05422222

VX21-121-105

2024-513754-29-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Enrollment: 210 estimated patients

Sex: All

Ages: 1 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene

Key Exclusion Criteria:

• History of solid organ, hematological transplantation, or cancer
• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
• Lung infection with organisms associated with a more rapid decline in pulmonary status

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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210 participants in 2 patient groups

Part A: VX-121/TEZ/D-IVA

Experimental group

Description:

Participants will receive VX-121/TEZ/D-IVA in the morning.

Treatment:

Drug: VX-121/TEZ/D-IVA

Part B: VX-121/TEZ/D-IVA

Experimental group

Description:

Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.

Treatment:

Drug: VX-121/TEZ/D-IVA