Inhaled Therapy Adherence and Outcomes to Kaftrio in Cystic Fibrosis

Brief summary (registry, in English)

Cystic fibrosis (CF) is a life-limiting and life-long genetic condition which requires intensive preventative treatment to manage the symptoms and progression of disease. While preventative treatments target the effects of cystic fibrosis, precision medicines target the underlying dysfunction of the cystic fibrosis transmembrane regulator (CFTR) protein at a cell level. The first of these expensive precision medicines also known as modulator therapies, Ivacaftor, was shown to be highly effective in clinical trials with an increase of over 10% in lung function. Real-world studies showed an increase of only 6% and a return to baseline lung function by year five of treatment. Preventative therapies were continued during the Ivacaftor clinical trials whereas there is real world evidence of declining inhaled preventative therapy use following Ivacaftor initiation. This is a potential explanation for the efficacy-effectiveness gap. The first study in the National Efficacy Effectiveness Modulator Optimisation (NEEMO) programme is exploring this (REC ref: 21/HRA/4940, IRAS 301975). Ivacaftor/Tezacaftor/Elexacaftor is the most recent modulator available, commissioned in the UK (United Kingdom) in 2020, and suitable for around 90% of people with cystic fibrosis. It is not yet known if the efficacy effectiveness gap seen with Ivacaftor also exists for Ivacaftor/Tezacaftor/Elexacaftor. There is also uncertainty about the continued need for preventative inhaled therapy alongside the prescription of Ivacaftor/Tezacaftor/Elexacaftor. This second study in the NEEMO programme is a cohort, observational study and will explore adherence to inhaled preventative therapies in adults with cystic fibrosis before and after commencing Ivacaftor/Tezacaftor/Elexacaftor, and in those not prescribed Ivacaftor/Tezacaftor/Elexacaftor. It will also look at the relationship between adherence to preventative inhaled therapy and outcome for adults with CF taking Ivacaftor/Tezacaftor/Elexacaftor. The analysis will use routinely collected pseudo anonymised data from the CFHealthHub learning health system (CFHealthHub), alongside anonymised data from the CF registry and routinely collected clinical data.

What you commit to

• Study type: Observational
• Participants enrolled: 1385
• From 27 Jul 2022 to the primary completion date, 31 Oct 2024
• 1 site in 1 country
• Visits and tests: ask the trial team, the registry does not list them

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Who cannot take part

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Who the study is looking for

Age in the registry: 16 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

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