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

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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

NCT05422222
2021-005930-40 (EudraCT Number)
VX21-121-105

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)

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

Trial contacts and locations

34

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Central trial contact

Medical Information

Data sourced from clinicaltrials.gov

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