A new study will assess the real-world effects of Vertex Pharmaceutical‘s Trikafta, a triple combo of elexacaftor, tezacaftor, and ivacaftor approved by the U.S. Food and Drug Administration (FDA) for treating up to 90% of all people with cystic fibrosis (CF).
The $3.63 million study (3.06 million euros), called RECOVER, will assess the impact of Trikafta (known as Kaftrio in Europe) on the everyday lives of people with CF, rather than in controlled clinical trial conditions.
RECOVER is led by researchers at the RCSI University of Medicine and Health Sciences, Ireland, in collaboration with 16 clinical and university sites in the U.K., Europe and North America.
The funds were awarded by the Cystic Fibrosis Foundation in the U.S. ($3.38 million, or 2.85 million euros), the Cystic Fibrosis Trust in the U.K. ($132,780, or 112,000 euros), and Cystic Fibrosis Ireland ($118,550, or 100,000 euros).
“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,” Paul McNally, MD, said in a press release. McNally is associate professor of pediatrics at RCSI, and lead researcher for 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,” said McNally, who also is a respiratory consultant at Children’s Health Ireland.
The project, which will start patient recruitment in August, will assess the clinical outcomes of CF patients across centers in the U.K. and Ireland over a period of two years.
Besides examining routine health parameters, researchers will evaluate parameters not included in the AURORA clinical trials that led to the approval of the therapy in the U.S., including imaging, functional, biological and quality of life measurements.
Trikafta is the most recent CF therapy to be approved in the U.S as a treatment for people 12 and older who have either two F508del mutations or one F508del mutation and one minimal function mutation in the CFTR gene, which causes the disease.
Approval is pending in Europe after the Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion for Kaftrio in combination with Kalydeco (ivacaftor) for the same CF conditions as in the U.S.
“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,” said Philip Watt, CEO, Cystic Fibrosis Ireland.
Lucy Allen, director of research at the Cystic Fibrosis Trust, added: “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,” she said. “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.”
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