A Study Evaluating the Safety of Elexacaftor Combination Therapy
Research type
Research Study
Full title
A Phase 3b Open-label Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects
IRAS ID
273613
Contact name
Jamie Duckers
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2019-003455-11
Duration of Study in the UK
1 years, 2 months, 27 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 ELX used in combination with TEZ and IVA in patients with Cystic Fibrosis who are 12 years of age or older, and who have certain mutations in their CF genes.This study will evaluate the safety of 48 weeks of treatment with ELX/TEZ/IVA in subjects with CF who have an F/F (homozygous for F508del) mutation ( a structural gene change ) genotype who complete a parent study (VX18-445-109). Data from this study are intended to enrich the body of evidence showing that ELX/TEZ/IVA is safe and well tolerated in subjects with F/F genotypes.
The total study duration is approximately 52 weeks (from the first dose of study drug in this study), including a Treatment Period of 48 weeks and a 4 week Safety Follow up Visit.Results Summary
Lay Summary Results for VX19-445-115, A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects
This study involved cystic fibrosis (CF) participants who were 12 years old or older. It took place at 29 locations in 4 countries: Australia, Belgium, Germany and the UK. 172 people took part in this study.
The study had 2 parts. In Part A participants got 200 milligrams of Elexacaftor (ELX) once a day, 100 milligrams of Tezacaftor (TEZ) once a day and 150 milligrams of Ivacaftor (IVA) twice a day for 48 weeks. In Part B participants got 200 milligrams of ELX once a day, 100 milligrams of TEZ once a day and 150 milligrams of IVA twice a day for 86 weeks. Out of the 172 patients from Part A, 159 patients completed the study, and only 50 patients participated in Part B of the study.
Some people had side effects from the study medication. Common side effects were headache, fever, worsening of lung symptoms like coughing or shortness of breath, runny nose and sore throat.
The purpose of this study was to look at how CFTR corrector drugs affect important health benefits and risks in people with cystic fibrosis who have two F508del mutations. For more information, detailed results are publicly available at https://gbr01.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.clinicaltrials.gov%2F&data=05%7C02%7Cgmsouth.rec%40hra.nhs.uk%7Cd8b4b9c638b6428cd0cb08dd5d955395%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638769618121719429%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=uOQdPN6eCMDVTN93N1AqiyvuWYlXPabEkb7fs0A%2BxoI%3D&reserved=0, search NCT04362761 or VX19-445-115.REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
19/NW/0716
Date of REC Opinion
17 Jan 2020
REC opinion
Further Information Favourable Opinion