Real World Clinical Outcomes With Novel Modulator Therapy Combinations in People With CF (RECOVER)

• Status: Recruiting
• Trial end date: 2024-07-01
• Target enrollment: 0
• Participant gender: All

Summary

RECOVER is a prospective, multicenter observational study designed to measure the real world clinical effectiveness of elexacaftor, tezacaftor and ivacaftor triple combination therapy (Kaftrio) in people with cystic fibrosis over a two year 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 children aged six years and above over a period of two years. The first phase of the study will commence in 2020, recruiting children 12 years and older who have started on clinical treatment with Kaftrio.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Royal College of Surgeons, Ireland
• Collaborators: Cystic Fibrosis Registry of Ireland; Erasmus Medical Center; Imperial College London; Medizinische Hochschule Brandenburg Theodor Fontane; Queen's University, Belfast; Teagasc; The Hospital for Sick Children; University College Dublin; University of Limerick

Criteria

Inclusion Criteria:

Inclusion criteria People with CF aged 12 years and over: 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 Kaftrio 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 Kaftrio. Participants will receive treatment only through prescription by their physician through usual clinical treatment pathways.

Children aged 6-11 years: Children aged 6-11 years will be included in the study only if and when Kaftrio is licenced, approved and funded for this age group. 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 Kaftrio 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 Kaftrio. Participants will receive treatment only through prescription by their physician through usual clinical treatment pathways.

Subjects on Kaftrio 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 Kaftrio 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 (people with CF aged 12 years and over, children aged 6-11 years and subjects on Kaftrio) must be taking the full dose of Kaftrio (in accordance with the age appropriate posology in the SmPC).

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 Kaftrio 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.

Any contraindication to Katrio treatment as per the local approved SmPC.

Severe hepatic impairment.

Pregnant and breastfeeding women.