What is RECOVER?
RECOVER (Real World Clinical Outcomes with Novel Modulator Therapy Combinations in People with CF) is a new non interventional study that aims to look at the ‘real world’ impact of the new triple combination modulator, Kaftrio®, in people with CF.
Why do we need to do this study?
Although the clinical trials have proven that triple combination therapy is very effective for specific groups of people with CF, they are very controlled and highly monitored studies. Often things can be different in the everyday life, so it is really important to look at this also to prove that new medicines are effective. In this real world study, we have an opportunity to look at a wider array of outcomes that are important to people with CF.
Why is this study important?
New medications such as the Kaftrio® involve a significant investment on behalf of the state, and it is really important to be able to prove that the treatment is beneficial and value for money. Real world studies like RECOVER help to gather this information. Nowadays, there are lots of new expensive therapies for rare diseases, all competing for limited health budgets. Providing high quality evidence for CF treatments will help us to maximise the likelihood of these treatments being funded now and into the future for people with CF.
What does RECOVER involve?
RECOVER will collect information both on routine outcomes already collected as part of standard care, such as lung function, infections, nutrition etc. but also novel and uncommonly used outcome measures. We will employ detailed measures of lung health such as lung clearance index (LCI), CT scans, exhaled nitric oxide and sputum inflammation. Abdominal complaints are common in CF. We will measure abdominal symptoms, bowel inflammation, pancreatic status and markers of liver disease. We want to also examine how people with CF remember to take Kaftrio®, and all other CF medicines after Kaftrio® has been started. Other aspects of RECOVER include looking at nasal inflammation, quality of life and how different genes might affect how different people respond to these new therapies. As part of the sinus disease sub study, nasal swabs will be collected, as well as nasal lavage on patients aged 6-11 who routinely perform sinus rinses at home. Sinus MRIs will be performed on a small cohort of 20 participants in Children’s Health Ireland (CHI). Breath sample collection will take place on a small cohort of 50 participants aged 6-11 in Guy’s and St. Thomas NHS Foundation Trust and CHI. The psychology sub-study will be performed at 12-month visit for participant’s aged 12-17 years of age.
Where is RECOVER taking place?
RECOVER is a truly international study. Funding for RECOVER comes predominantly from the US CF Foundation, with co-funding from CF Ireland and the UK CF Trust. The clinical sites for RECOVER are in Ireland and the UK (adult and paediatric), and we are working with collaborators across the UK and Ireland, Canada, Germany and the Netherlands. RECOVER does not have any relationship with Vertex Pharmaceuticals, the manufacturer of the Kaftrio®.
Who is involved?
RECOVER involves a team of leading CF specialists, scientists, data experts and representatives of people with CF. The team is lead by Paul McNally (Dublin) and Jane Davies (London), both paediatricians looking after people with CF. The team includes consultants in each of the clinical sites.
Where do I get more information?
You can get more information about RECOVER by asking your CF nurse or consultant. The research team at your centre and one of the CF consultants are involved in the study and have more information. There are information leaflets for adults, parents of children, adolescents and young children.
Will I receive my results?
Results collected during the course of RECOVER are for research purposes only and will not direct clinical care. Your CF team at your site may be able to share certain results with you throughout the course of the study, for example sweat chloride levels and LCI results, however this will be subject to local policies.
Additionally, for further information and/or queries please contact us at info@RECOVERCF,