Inhaled therapy adherence and outcomes to Kaftrio in cystic fibrosis
Research type
Research Study
Full title
Exploring adherence to preventative inhaled therapy and outcomes for people with cystic fibrosis who take Ivacaftor/Tezacaftor/Elexacaftor.
IRAS ID
313840
Contact name
Tracey Daniels
Contact email
Sponsor organisation
Sheffield Teaching Hospitals NHS Trust
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
22060, STH reference
Duration of Study in the UK
4 years, 6 months, 31 days
Research summary
Ivacaftor/Tezacaftor/Elexacaftor is a medication called a modulator which is used to help lung and other cells work better for people with cystic fibrosis (CF); a life-long and life-limiting condition. Clinical trials of Ivacaftor/Tezacaftor/Elexacaftor showed an increase of over 10% in lung function.
A gap between the improvements seen in clinical trials and improvements in everyday use (an efficacy effectiveness gap) was seen for Ivacaftor, the first available modulator. A 10.6% improvement in lung function was seen in clinical trials of Ivacaftor but only 6.0% within real-world data. There are no data that shows whether an efficacy-effectiveness gap exists for Ivacaftor/Tezacaftor/Elexacaftor .
The aim of this study is to understand the real-world effectiveness of Ivacaftor/Tezacaftor/Elexacaftor and to identify any efficacy-effectiveness gap. Changes in the preventative inhaled treatments that people take before and after starting Ivacaftor/Tezacaftor/Elexacaftor will be explored to understand whether this may affect lung function improvement.
Participants will be adults with CF in the UK, who attend one of the CF centres who have participated in the CFHealthHub learning health system, and who have CF registry consent, allowing anonymised data to be used for future research. Some people will also have CFHealthHub consent, allowing pseudo anonymised adherence data to be used for future research. Routinely collected clinical and adherence data will be analysed from twelve months before starting Kaftrio and for up to five years after.
This study is important because lung function is key to length of life and how well people with CF are. Making a choice about taking burdensome inhaled treatments is difficult because there is no information about how taking less inhaled treatment when on Ivacaftor/Tezacaftor/Elexacaftor might affect lung function. This study is important to policy makers as Ivacaftor/Tezacaftor/Elexacaftor and inhaled treatments are expensive and it is important that the best benefits of Ivacaftor/Tezacaftor/Elexacaftor are realised.
REC name
North of Scotland Research Ethics Committee 2
REC reference
22/NS/0090
Date of REC Opinion
27 Jun 2022
REC opinion
Favourable Opinion