Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Launched by VERTEX PHARMACEUTICALS INCORPORATED · Apr 24, 2023

Keywords

ClinConnect Summary

This study is testing a long-term use of a three-drug combination (vanzacaftor/tezacaftor/deutivacaftor) called VNZ/TEZ/D-IVA in people with cystic fibrosis. It’s a Phase 3, open-label trial, meaning all participants receive the medicine and both they and their doctors know what is being given. About 174 people aged 1 year and older, who have completed a prior related study, will be invited to join. The treatment is given once daily, with the dose based on age and weight. Part A covers the initial treatment period, and Part B offers an extended treatment for some participants, potentially up to 196 weeks in total. People with significant liver disease, a history of cancer, or a transplanted organ are not eligible.

Safety is the main focus at first, looking at the number of participants who have adverse events (side effects). Researchers will also look at how well the treatment works over time, using measures like sweat chloride (a test of CFTR protein function), lung function (ppFEV1), and how often people have lung infections or CF-related hospital visits. Growth and nutrition are also tracked (things like BMI and weight/height for age), along with quality-of-life questions. The study is being conducted at many sites around the world and is sponsored by Vertex Pharmaceuticals. Enrollment is by invitation and the trial is planned to run through 2029; results are not yet available.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• Parts A and B:
• • • Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)
• Part B:
• -Meets at least 1 of the following criteria:
• • Completed study drug treatment in Part A
• • Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A
• Key Exclusion Criteria:
• • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
• • History of solid organ, hematological transplantation, or cancer
• • History of drug intolerance in the parent study
• • Other protocol defined Inclusion/Exclusion criteria may apply.