Table of contents
- Overview of the trials
- Who the studies include
- What the trials are measuring
- Trial phases and study size
- Main studies listed
- Important terms explained
Overview of the trials
The clinical trials for Vanzacaftor in the provided data all focus on cystic fibrosis and are designed as interventional studies, meaning the researchers give a treatment and then observe what happens.[1][2][3]
All three trials are in Phase 3, which is a later stage of clinical research used to study a treatment in larger groups of people.[1][2][3]
The studies are authorised and include participants from very young children to adults, depending on the trial.[1][2][3]
Who the studies include
One trial is for people with cystic fibrosis who are 1 year of age and older.[1]
A second trial includes subjects with cystic fibrosis without the age range being stated in the source data, and it has the largest enrolment among the listed studies.[2]
The third trial is focused on children with cystic fibrosis who are 1 through 11 years of age.[3]
These age groups show that the research is not limited to adults; it also includes children, which is important because treatment needs can differ by age.[1][3]
What the trials are measuring
The main goal in two of the studies is to assess safety and tolerability of long-term treatment, using adverse events, clinical laboratory values, ECGs, vital signs, and pulse oximetry.[1][2]
In simple terms, this means the researchers want to see whether the treatment can be used over time and whether it causes any important medical problems during the study.[1][2]
The child-focused study also measures PK parameters in Part A, which means it looks at how the treatment and its related substances behave in the body.[3]
That same study also checks safety and tolerability in both Part A and Part B, and Part B follows safety through Week 24.[3]
Trial phases and study size
All three trials are Phase 3 studies, so they are testing the treatment in a larger and more practical patient setting than earlier-stage research.[1][2][3]
The enrolment numbers in the source data are 134, 844, and 122 participants, showing different study sizes and likely different research questions.[1][2][3]
The largest study is the one with 844 participants, which suggests a broad safety and tolerability evaluation in cystic fibrosis.[2]
Main studies listed
The first study is titled Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Subjects 1 Year of Age and Older.[1]
Its brief summary says the purpose is to evaluate the long-term safety and tolerability of VNZ/TEZ/D-IVA.[1]
The second study is titled A Phase 3 Study of VX-121 Combination Therapy in Subjects With Cystic Fibrosis.[2]
Its brief summary says it evaluates the long-term safety and tolerability of VX-121/tezacaftor/deutivacaftor in subjects with cystic fibrosis.[2]
The third study is titled Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis Subjects 1 Through 11 Years of Age.[3]
This study has two parts: Part A evaluates PK and safety, while Part B focuses on safety and tolerability through Week 24.[3]
The source data also lists several treatment names and product labels, including VX-121/VX-661/VX-561, Alyftrek, Kalydeco, Kaftrio, and VX-445/VX-661/VX-770, but the key point of the trials is their clinical evaluation in cystic fibrosis.[1][2][3]
Important terms explained
Adverse events are any unwanted health problems that happen during a study, whether or not they are caused by the treatment.[1][2][3]
Clinical laboratory values are results from blood or other tests that help researchers watch for changes in health.[1][2][3]
ECG, or electrocardiogram, is a test that records the heart’s electrical activity.[1][2][3]
Vital signs are basic health measures such as pulse and blood pressure.[1][2][3]
Pulse oximetry is a quick test that checks oxygen levels in the blood.[1][2][3]
Pharmacokinetics, often shortened to PK, describes how the body handles a treatment over time.[3]
