Badanie bezpieczeństwa i skuteczności VX-828, Deutivacaftoru i Tezacaftoru u dorosłych z mukowiscydozą

Baseline Assessment

After joining the study you attend a baseline visit where the research team records your medical history, measures your vital signs (blood pressure, pulse), performs a standard 12‑lead ecg (electrocardiogram) to check heart rhythm, takes blood samples for laboratory tests, conducts a sweat chloride test (measures salt level in sweat) and a lung function test that determines the amount of air you can exhale in one second (ppfev1). you also complete a CFQ R Questionnaire that asks about respiratory symptoms.

Randomization and assignment

Based on a computer‑generated schedule you are assigned to one of four treatment groups. Each Group receives a specific combination of study tablets or matching placebos, but you will not know which one you receive because the study is double‑blind.

Start of study medication

You begin taking the study medication daily for 28 consecutive days. The tablets are taken by mouth (oral) and should be swallowed with water.

If you are in the combination arm that includes tezacaftor, the daily regimen is:

VX-828 20 mg tablet, deutivacaftor 250 mg film‑coated tablet, and tezacaftor 100 mg tablet (or the equivalent fixed‑dose tablet).

If you are in the arm without tezacaftor, the daily regimen is:

VX-828 20 mg tablet and deutivacaftor 250 mg film‑coated tablet.

If you are assigned to a placebo Group, you take tablets that look identical but contain no active drug.

Daily dosing routine

Each day you take the assigned tablets at roughly the same time, preferably with food to reduce stomach upset.

You keep a simple diary noting the date and time of each dose and any new symptoms or side effects you notice.

Interim safety checks

During the 28‑day treatment period you may be asked to visit the clinic or contact the study staff for Brief safety checks, such as reporting adverse events (aes – any unwanted medical occurrence) or answering questions about how you feel. These checks are short and do not require additional medication.

Day 28 final evaluation

On day 28 you return for a final evaluation. The same assessments performed at baseline are repeated: vital signs, ecg, laboratory tests, sweat chloride test, lung function test (ppfev1), and the CFQ R Questionnaire.

The purpose is to compare the results with the baseline values to see any change after 28 days of treatment.

End of treatment and follow‑up

After the day 28 visit you stop taking the study tablets. The research team reviews all safety information and may schedule a short follow‑up contact a few weeks later to ensure no delayed adverse events occur.