Table of contents
- Trial overview
- Who is being studied
- What the trials measure
- Trial phases and status
- Trial designs and treatment groups
- Key patient points
Trial overview
The available studies of Vanzacaftor are all in people with cystic fibrosis, and all are Phase 3 interventional trials.[1][2][3]
These trials are authorised and are designed to look mainly at long-term safety and tolerability, with one study also looking at pharmacokinetics, which means how the treatment is handled by the body.[1][3]
Who is being studied
One trial includes subjects 1 year of age and older, showing that the research covers both children and adults with cystic fibrosis.[1]
Another trial is focused on subjects 1 through 11 years of age, which means it is aimed at younger children with cystic fibrosis.[3]
A third Phase 3 study includes subjects with cystic fibrosis in a broader group and has the largest planned enrollment in the source data.[2]
What the trials measure
The main safety outcomes include adverse events, clinical laboratory values, ECGs, vital signs, and pulse oximetry.[1][2][3]
Adverse events are any unwanted medical problems seen during a study, whether or not they are caused by the treatment.[1]
ECGs are heart tests, vital signs are basic body measurements, and pulse oximetry checks blood oxygen levels.[1][2][3]
One study also measures PK parameters, a short name for pharmacokinetic data, which describes how the body absorbs and processes the treatment and its related metabolites.[3]
Trial phases and status
All three trials are in Phase 3, which is a later stage of clinical research used to study treatment performance in larger groups of patients.[1][2][3]
Each study is listed as Authorised, so they are approved to move forward according to the source data.[1][2][3]
The planned enrollment numbers are 134, 844, and 122 participants, showing that the studies vary in size.[1][2][3]
Trial designs and treatment groups
All listed studies are interventional, meaning researchers give study treatments and then observe the results.[1][2][3]
The source data lists several treatment forms, including film-coated tablets and granules, and also includes product names such as Alyftrek, Kalydeco, and Kaftrio in the intervention lists.[1][2][3]
In the study titles and summaries, the treatment is described as Vanzacaftor/tezacaftor/deutivacaftor or VX-121/TEZ/D-IVA, which appears as the main triple-combination study treatment in the source data.[1][2][3]
Key patient points
These trials are focused on cystic fibrosis, not on other diseases.[1][2][3]
The main question is whether long-term treatment can be used safely and well over time in the studied age groups.[1][2][3]
One study also adds body-processing data, which can help researchers understand how the treatment behaves in younger children and other participants.[3]
