Elexacaftor/Tezacaftor/Ivacaftor: The intervention is the same for both study groups. In addition to all the assessments in the standard arm, the advanced arm subjects will undergo spirometry controlled CT, nasal lavage and sputum sample collection.
Study summary
RECOVER is a prospective, multicenter observational study designed to measure the real world clinical effectiveness of elexacaftor/tezacaftor/ivacaftor (ETI) triple combination therapy in people with cystic fibrosis initially over a two year period, followed by a five year extension period. Measured outcomes include measures of lung function, lung inflammation, lung imaging, abdominal symptoms, gut inflammation, liver function, pancreatic exocrine function, nasal inflammation, quality of life and adherence to therapy. The study will examine outcomes in two cohorts, children aged six to eleven years and children and adults aged twelve and above.
Eligibility
Sex
ALL
Min age
6 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
Participants may only be selected for inclusion in RECOVER if they have been independently determined by their treating physician to be suitable for treatment with ETI in compliance with the official marketing authorization and summary of product characteristics (SPC).
The decision to include participants in the study is independent of decision to prescribe ETI.
Participants will receive treatment only through prescription by their physician through usual clinical treatment pathways.
Subjects on ETI In exceptional circumstances where baseline clinical data has been collected prior to the start of treatment either through clinical care or ethically approved research projects (including a cohort of subjects initially recruited to this study on the understanding that it was a non-regulated observational study) subjects already receiving ETI may be recruited to this study and undergo on-treatment visits. Any additional patient data can only be added with written informed consent from the patients/parents concerned.
All subjects must have a signed informed consent form and/or signed assent form when appropriate, as determined by the subjects age and individual site and country standards.
Male and female participants of childbearing potential must agree to adhere to contraception requirements as detailed in the local ETI SmPC and in line with the standard of care.
Exclusion Criteria:
Patients not willing to comply with study procedures or assessments.
Individuals on clinical trials of investigational CFTR modulators.
Clinical instability at baseline assessments. Subjects undergoing an active exacerbation and at the beginning of their treatment should be excluded from the study as this is likely to skew the data.
Primary outcome measure(s)
Change in LCI on treatment with ETI in children and adults with CF. — 84 month period Measured using multiple breath washout (MBW)
Trial sites (7)
Facility
City
Region
Status
Children's Health Ireland at Crumlin
Dublin
Leinster
University Hospital Limerick
Limerick
Limerick
Children's Health Ireland at Temple Street
Dublin
Ireland
St. Vincent's University Hospital
Dublin
Ireland
Children's Health Ireland at Tallaght
Tallaght
Ireland
Royal Belfast Hospital for Sick Children
Belfast
United Kingdom
Royal Brompton Hospital
London
United Kingdom
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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