Cystic fibrosis study to investigate real-world effects of new triple combination drug

Dublin, Ireland, Thursday, 30 July 2020 – Researchers at RCSI University of Medicine and Health Sciences are leading a new €3m study to investigate the real-world effects of the newest cystic fibrosis medications as they become available for people with CF.

The research will look at how a new triple combination cystic fibrosis drug, Kaftrio, will affect people in their day to day lives, rather than in controlled clinical trial conditions. Kaftrio (known as TriKafta in the US) is a successor to Orkambi.

The project, called RECOVER, has been awarded €2.85 m from Cystic Fibrosis Foundation (US), €112 thousand from Cystic Fibrosis Trust (UK) and €100 thousand from Cystic Fibrosis Ireland.

In recent years, a number of promising new medications have been developed that treat the underlying genetic defect in cystic fibrosis. The most recent triple combination cystic fibrosis drug, Kaftrio, has demonstrated striking improvements in clinical trials of people with the most common form of cystic fibrosis.

Pending final European Medicines Agency approval, expected in the next number of weeks, these new medications will be provided for use in the majority of people with cystic fibrosis in Ireland and the UK as part of an existing agreement between the manufacturer and the respective states.

Starting in August 2020, the study will recruit patients and begin to examine clinical outcomes over a two year period across cystic fibrosis centres in Ireland and the UK. In addition to collecting routine health measurements, they will study detailed imaging, functional, biological and quality of life measurements that could not be collected in clinical trials.

“Our study will allow us to discover in detail how this powerful new treatment affects the health and everyday lives of people with cystic fibrosis, to understand why different people might respond differently to the drug and to gain insight into how this treatment might affect the very significant treatment burden that people with cystic fibrosis currently endure,” said Professor Paul McNally, Associate Professor of Paediatrics at RCSI, Respiratory Consultant in Children’s Health Ireland and lead researcher on the RECOVER study.

“Clinical trials occur under very controlled conditions and exclude many sick people with cystic fibrosis. ‘Real-world’ studies, such as RECOVER, are essential to confirm the trial findings and examine in-depth effects on the everyday lives all people with cystic fibrosis.”

In partnership with the Irish and UK cystic fibrosis registries, RECOVER is led by an Irish research team collaborating with researchers in 16 clinical and university sites in the UK, Europe and North America.

Philip Watt, CEO, Cystic Fibrosis Ireland commented: “Cystic Fibrosis Ireland is proud to support this major research study that will provide much new detailed data on the impact of new and innovative CF medications across Europe and North America.”

Dr Lucy Allen, Director of Research at the Cystic Fibrosis Trust, said: “The data from the clinical trials have shown us that Kaftrio will make a significant difference to people’s lives. However, as CF is a complex condition affecting many parts of the body it is important to understand the full impact of this drug. In the short term the results of the RECOVER study may speed up access to Kaftrio around the world, in the longer term it will provide information on tailoring care for each individual with cystic fibrosis”.

Led by RCSI, the collaborating institutions include, Imperial College, London; Children’s Health Ireland (at Crumlin, Tallaght, Temple Street and Connolly); National Children’s Research Centre; St Vincent’s University Hospital, Dublin; University Hospital Limerick; Belfast City Hospital; Royal Belfast Hospital for Children; Royal Brompton Hospital, London; CF Registry of Ireland, Dublin; UK CF Registry; The Hospital for Sick Children (SickKids), Toronto; Eramsus MC, University Medical Centre, Rotterdam; Brandenburg Medical School, Germany; Queen’s University Belfast; Teagasc Food Research Centre, Cork; Hospital for Sick Children, Toronto.

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Notes to Editors

• Professor Paul McNally Associate Professor of Paediatrics at RCSI University of Medicine and Health Sciences is available for interview.
• Photography is available on request

About Cystic Fibrosis

• Cystic fibrosis (CF) is an inherited chronic disease that primarily affects the lungs and the digestive system. A defective gene causes the body to produce unusually thick sticky mucus that clogs the lungs and obstructs the pancreas, stopping natural enzymes from enabling the body to break down and absorb food.
• Ireland has the highest incidence of cystic fibrosis in the world, with almost 1,400 people diagnosed with the disease here. Ireland also has some of the more severe forms of the disease.
• Approximately one in 19 are thought to carry one copy of the altered gene that causes CF.
• People with CF experience a range of symptoms including frequent lung infections, persistent coughing, wheezing and shortness of breath, along with poor growth and weight gain in spite of a good appetite. Over time people with CF accumulate significant damage and scar tissue in their lungs which leads to end-stage lung disease requiring a double lung transplant if possible.
• According to the Cystic Fibrosis Registry of Ireland Annual Report 2018, between 2010 and 2018, an average of 40 individuals were diagnosed with CF each year.
• In 2018, there were 140 individuals with CF aged 40 years or older.
• In 2018, the median age of a person with CF was 21 years. In other words, half of the 1,239 individuals registered were under 21 years of age, and the other half were aged 21 years or older. However, survivability for a growing number is now predicted into their 30s and 40s and beyond.
• The counties with the largest numbers of people with CF are Dublin (327), Cork (167), Limerick (73), Tipperary (60) and Galway (59). In 2018, there was a total of 1,129 hospitalisations for 529 people with CF.

About RCSI University of Medicine and Health Sciences

• Ranked number one globally for Good Health and Well-being in the Times Higher Education (THE) University Impact Rankings 2020, RCSI University of Medicine and Health Sciences is an international not-for-profit university, with its headquarters in Dublin.
• RCSI is exclusively focused on education and research to drive improvements in human health worldwide. It is among the top 250 universities worldwide in the THE World University Rankings (2020) and its research is ranked first in Ireland for citations. RCSI has been awarded Athena Swan Bronze accreditation for positive gender practice in higher education.
• Visit the RCSI MyHealth Expert Directory to find the details of our experts across a range of healthcare issues and concerns. Recognising their responsibility to share their knowledge and discoveries to empower people with information that leads them to better health, these clinicians and researchers are willing to engage with the media in their area of expertise.

About Cystic Fibrosis Ireland

• Cystic Fibrosis Ireland (CFI) is a leading national organisation dedicated to improving treatment and facilities for people with CF and their families across Ireland. The organisation is dedicated solely to the well-being of people with CF in Ireland.
• Its mission is to assist the development of the means to cure and control CF, to promote the interests and welfare of people with CF in Ireland and to assume advocacy for them and their needs resultant from their having CF.
• It does this through providing information and education, advice and advocacy to people and their families, undertaking medical and scientific research, advancing the development of lung transplantation in Ireland, and offering grant assistance to patients.
• CFI also funds a number of specialist CF clinical posts as well as providing capital funding for the development of state-of-the-art CF units around the country, including dedicated in-patient, day care and out-patient facilities.
• CFI comprises 18 branches and one regional branch and is overseen by a national executive committee, which includes representation from people with CF and their families. For more, visit http://www.cfireland.ie

About the Cystic Fibrosis Trust

• The Cystic Fibrosis Trust is the only UK-wide charity dedicated to fighting for a life unlimited for everyone affected by cystic fibrosis
• For confidential advice, support and information on any aspect of cystic fibrosis, including help with financial support contact the Cystic Fibrosis Trust helpline on (+44) 0300 373 1000 or 020 3795 2184
• The work we do is only made possible by the generous donations from our supporters. Visit http://www.cysticfibrosis.org.uk to find out more about cystic fibrosis, the work of the Trust and how you can help our fight for a life unlimited.