Phase 4 Decentralised Pilot Study With Wearable Technology in CF
Research type
Research Study
Full title
A Phase 4 Decentralized Pilot Study With Wearable Technology in Cystic Fibrosis Subjects 18 Years of Age and Older Taking Commercial Elexacaftor/Tezacaftor/Ivacaftor
IRAS ID
297635
Contact name
Nicholas Simmonds
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Duration of Study in the UK
0 years, 3 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. Trikafta/Kaftrio (ELX/TEZ/IVA) regimen is the first medicine to demonstrate clinical benefit in patients with a single F508del allele, regardless of the mutation of the second allele. A pivotal Phase 3 program in CF subjects 12 years of age or older demonstrated that ELX/TEZ/IVA provides substantial improvements in lung function, CFTR function and was generally safe and well tolerated with a low rate of treatment discontinuation.
The purpose of this pilot study is to evaluate the performance of wearable technology in CF subjects taking commercial ELX/TEZ/IVA, as well as to conduct a fully decentralized study in this population, leveraging a technology platform that allows for fully virtual participation.REC name
London - Chelsea Research Ethics Committee
REC reference
21/PR/0599
Date of REC Opinion
3 Aug 2021
REC opinion
Further Information Favourable Opinion