In short
Clinical trials are studying Vanzacaftor in people with cystic fibrosis. These studies look at long-term safety, tolerability, and treatment effects in children and adults, including patients 1 year of age and older. The trials are in Phase 3 and focus on how well the treatment is used over time.
Key points
- Clinical trials of Vanzacaftor are being done in people with cystic fibrosis. All listed studies are Phase 3 and are authorised. The trials include children and adults, including people 1 year of age and older and a study for ages 1 through 11 years. The main goals are to assess long-term safety and tolerability, and one study also measures pharmacokinetic data. Researchers track outcomes such as adverse events, lab tests, ECGs, vital signs, and oxygen levels.
Trial overview
The available studies of Vanzacaftor are all in people with cystic fibrosis, and all are Phase 3 interventional trials.
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.
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.
Another trial is focused on subjects 1 through 11 years of age, which means it is aimed at younger children with cystic fibrosis.
A third Phase 3 study includes subjects with cystic fibrosis in a broader group and has the largest planned enrollment in the source data.
What the trials measure
The main safety outcomes include adverse events, clinical laboratory values, ECGs, vital signs, and pulse oximetry.
Adverse events are any unwanted medical problems seen during a study, whether or not they are caused by the treatment.
ECGs are heart tests, vital signs are basic body measurements, and pulse oximetry checks blood oxygen levels.
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.
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.
Each study is listed as Authorised, so they are approved to move forward according to the source data.
The planned enrollment numbers are 134, 844, and 122 participants, showing that the studies vary in size.
Trial designs and treatment groups
All listed studies are interventional, meaning researchers give study treatments and then observe the results.
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.
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.
Key patient points
These trials are focused on cystic fibrosis, not on other diseases.
The main question is whether long-term treatment can be used safely and well over time in the studied age groups.
One study also adds body-processing data, which can help researchers understand how the treatment behaves in younger children and other participants.