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Journal of Cystic Fibrosis ; 20:S51, 2021.
Article in English | EMBASE | ID: covidwho-1368824


Background: Real-world, post-approval studies contribute significantly to the evidence surrounding the impact of new treatments, including CFTR modulators, but can be complex undertakings. Elexacaftor-tezacaftor-ivacaftor (ETI) was approved by EMA sooner than expected in August 2020 during a global pandemic. Method: RECOVER, a multi-centre, post-approval study examining the impact of ETI, and conducted in 8 clinical sites in Ireland and the UK over 2 years, examines important outcomes in children and adults prescribed ETI. The study will be conducted in 2 phases in line with ETI approval: 12+ and 6–11. In addition to routine data collected as part of normal care, key RECOVER endpoints include lung clearance index (LCI), spirometry-controlled CT, treatment adherence, GI symptoms, inflammation, liver disease markers, nasal inflammation and nitric oxide metabolism. Results: To date, 96 participants (56% female) out of a target of 137 in people with CFaged 12 and above, have been recruited (predominantly 12–18yrs to date). Recruitment and sample collection has been impacted by the effect of COVID-19 on CF care and CF centre attendance. Key challenges have included: Sputum collection (risks of induction and non-sputum producing participants) and coordination of study activities with limited clinic attendance. Despite this, key baseline data, prior to commencing treatment, has been successfully collected on the majority of participants to date. For subjects recruited to date, 56% have F508del/F508del and 44% F508del/minimum function mutations, mean age is 16.1 years, mean FEV1 83.6% (23–111%), mean LCI 12.2 (6.9–24.3). Recruitment and data collection is ongoing. Conclusion: Despite the impact of accelerated approval and COVID-19, we have been able to proceed with study initiation, recruitment and sample collection. Data from RECOVER and other international post-approval studies is likely to add significantly to our understanding of the impact of ETI on people with CF.

Journal of Cystic Fibrosis ; 20:S57, 2021.
Article in English | EMBASE | ID: covidwho-1361552


Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) has disrupted clinical trials. The European Cystic Fibrosis Society-Clinical Trials Network (ECFS-CTN) tracked disruption to CF trials via regular surveys to adult and paediatric clinics of member sites throughout 2020. We published preliminary results to May 2020 (doi: 10.1183/13993003.02114-2020). Here we report updated data to the end of 2020. Ongoing trials were heavily impacted up to May. Trial participant visits, new enrollment and monitoring visits were widely banned, with frequent home delivery of study drug (Table 1). From June to December, trial visits, new enrollment and onsite monitoring were mostly allowed, and home delivery of study drug dropped accordingly. The set-up of new trials was heavily impacted in March but recovered substantially from June on. Some sites had reduced staff available to work on CF trials. Table 1 presents how remote visits and measures were used by sites from June to December. Much of the early trial disruption resolved by end 2020;however, challenges remain to protect the progress of clinical research in CF during the pandemic.