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

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Jun 13, 2022

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment called VX-121/tezacaftor/deutivacaftor for young patients with cystic fibrosis (CF), a condition that affects the lungs and digestive system. The study aims to understand how the medication works in the body, how safe it is, and how well it helps improve the health of children aged 1 to 11 years old who have at least one specific type of mutation in their CF gene.

To participate, children must have stable cystic fibrosis and carry at least one "triple combination responsive" mutation, such as the F508del mutation. However, those with a history of major organ transplants, certain liver problems, or specific lung infections may not be eligible. Participants in the trial can expect to undergo regular health checks and monitoring while receiving the study medication. This trial is important because it may lead to better treatment options for children with CF, helping them manage their condition more effectively.

Gender

ALL

Eligibility 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.