* Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5
Research type
Research Study
Full title
A Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Through 5 Years of Age
IRAS ID
292548
Contact name
Jane Davies
Contact email
Sponsor organisation
Vertex Pharmaceuticals (Europe) Ltd
Eudract number
2020-002251-38
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 11 months, 30 days
Research summary
Cystic fibrosis (CF) is a lifelong disease resulting from changes (mutations) in the code for one gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 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. This study is being done to learn more about the safety and tolerability of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in patients with Cystic Fibrosis who are 2 to 5 years of age, and who have certain mutations in their CF genes.
This is a Phase 3, 2-part (Parts A and B)study where Part A primary objective will evaluate the PK of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC).Part A study will be conducted in United States only.
This Part B study will evaluate the safety and tolerability of ELX/TEZ/IVA in male and female CF subjects 2 through 5 years of age with certain gene mutations.
Participation in Part B could last for approximately 32 weeks with a screening period that will last up to 4 weeks,a study treatment period that will last up to 24 weeks and a follow-up period that will last up to 4 weeks. Subjects who are eligible will be offered the opportunity to enroll in an open-label extension (OLE) safety study.Lay summary of study results: This study involved cystic fibrosis (CF) participants who were 2 years old to 5 years old. It took place at 22 locations in 5 countries: Australia, Canada, Germany, the UK and the United States. 83 people took part in this study. The study had 2 parts. In Part A, participants who weighed more than or equal to 14 kilograms got 100 milligrams of Elexacaftor (ELX) once a day, 50 milligrams of Tezacaftor (TEZ) once a day and 75 milligrams of Ivacaftor (IVA) twice a day for 15 days. In Part B, participants who weighed more than or equal to 14 kilograms got 100 milligrams of ELX once a day, 50 milligrams of TEZ once a day and 75 milligrams of IVA twice a day for 24 weeks, and participants who weighed between 10 kilograms and 13 kilograms got 80 milligrams of ELX once a day, 40 milligrams of TEZ once a day and 60 milligrams of IVA every morning, and 59.5 milligrams of IVA every night for 24 weeks. 18 participants completed Part A and 74 participants completed Part B. 10 people participated in both parts of the study. Some people had side effects from the study medication. Common side effects were cough, fever, runny nose and vomiting. The purpose of this study was to look at the safety and tolerability of the combination of elexacaftor/tezacaftor/ivacaftor in children with CF. For more information, detailed results are publicly available at https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C02%7Capprovals%40hra.nhs.uk%7Ccd92a112b51d4b793c6508de7ab3dc7c%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C639083110100686875%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=Vls8sCMAqb7jgS7S179xD0%2FEFx3lntLE9bkiihGYEz8%3D&reserved=0, search NCT04537793 or VX20-445-111.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
22/NW/0259
Date of REC Opinion
17 Mar 2021
REC opinion
Further Information Favourable Opinion