Timberline Study - Cohort B2
Research type
Research Study
Full title
A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age
IRAS ID
1006429
Contact name
N/A N/A
Contact email
Sponsor organisation
Vertex Pharmaceuticals (Ireland) Limited
Eudract number
2021-005930-40
Clinicaltrials.gov Identifier
Research summary
The study is testing the activity of the study drug (VX-121/Tezacaftor/Deutivacaftor) in the body, the safety and how well it works for participants who are aged 1 to 11 years old with Cystic Fibrosis (CF). CF is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR). Changes in this gene can affect the amount of the protein made from this gene or how well the protein works. When there is not enough protein or it does not work properly, some fluids in the body become sticky and causes problems in the lungs and other organs. The aim of the study is to try and prevent the progression of CF by giving the study drug to participants earlier in life.
The UK will be participating in Cohort (group) B1 only which will evaluate participants aged 6 to 11 years old. They will participate in the study for up to 32 weeks and will receive the study drug for 24 weeks. The participants will visit a doctor approximately 10 times throughout the duration of their participant and will have phone contact with the doctor approximately 3 times. During the visits the participants will undergo a number of tests; Ophthalmologic (eye function) examination x2, Full physical examination x5, Weight and height x7, Vital signs x7, Pulse oximetry (amount of oxygen in the blood) x7, Standard 12-lead ECG x9, Pregnancy test (only females of childbearing potential) serum x6 and urine x1, Serum chemistry (amount of certain substances in the body) x7, Vitamin levels x4, Hematology (testing the blood) x7, HbA1c (blood sugar) x4, Coagulation (bloods clotting ability) x5, Urinalysis (urine test) x4, Multiple-breath washout x6, Spirometry (measurement of how well the lungs are working) x6, Sweat chloride (amount of chloride in sweat) x6, Blood biomarker sample x3, Fecal sample collection x6 and PK sampling (drug evaluation) x3.REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
22/WM/0239
Date of REC Opinion
20 Feb 2023
REC opinion
Further Information Favourable Opinion