Clinical Outcomes With Novel Modifier Therapy Combinations in CF
Research type
Research Study
Full title
Real World Clinical Outcomes With Novel Modifier Therapy Combinations in People with CF
IRAS ID
279116
Contact name
Jane Davies
Contact email
Sponsor organisation
Royal College of Surgeons Ireland
Clinicaltrials.gov Identifier
VIA Award No: 083, UK CF Trust
Duration of Study in the UK
3 years, 9 months, 1 days
Research summary
A new type of advanced treatment for people with cystic fibrosis (PWCF) called triple combination CFTR modulator therapy (TCMT), has shown promising results on important health outcomes in early clinical trials and will be approved for use in the clinic for many PWCF in the next few months. Clinical trials are highly monitored and tightly controlled, and regulators understand that it is important to also examine the use of new medicines in a ‘real world’ setting to ensure they help patients as they are supposed to.
We have designed a ‘real world’ study involving children and adults with CF across 8 paediatric and adult CF centres in the UK and Ireland in collaboration with CF registry of Ireland and the UK CF registry. We will be examining a wide range of well-established tests in these people, as well as some newer, more sensitive tests (not included in trials) looking at how this new treatment might affect health and quality of life in PWCF. We will be looking at how PWCF feel about this new treatment, how it affects their quality of life and whether it affects their need for, or ability to take, other CF treatments. We have designed RECOVER in such a way that we will recruit people of different ages and disease severity, collect a very comprehensive set of outcome measures and collect, catalogue and store important patient samples so that we can ensure maximum benefit from our patient's participation in this research project now and into the future.
REC name
South West - Cornwall & Plymouth Research Ethics Committee
REC reference
20/SW/0124
Date of REC Opinion
21 Sep 2020
REC opinion
Further Information Favourable Opinion