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

  • STATUS
    Recruiting
  • End date
    Jul 1, 2024
  • participants needed
    237
  • sponsor
    Royal College of Surgeons, Ireland
Updated on 31 May 2022
fibrosis
spirometry
ivacaftor
tezacaftor
elexacaftor

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.

Description

Our aim with RECOVER is to examine the clinical impact of Kaftrio on key clinical outcomes in people with CF in a real-world setting. For this study, in addition to some of the more traditional ways of monitoring clinical outcomes in people with CF such a standard lung function, nutrition, exacerbations and liver disease, we are proposing to include some novel outcome measures not typically used in clinical trials such as lung clearance index (LCI) and spirometry controlled chest CT.

By implementing an extensive study protocol that will include important outcomes in a number of areas of health in people with CF, and matching this to a comprehensive biosample collection plan, we will have the power to gain important insight into how Kaftrio works, and what impact it has on rescue of CFTR function in this group of people.

Data on the following outcomes will be collected during the study:

Lung Clearance Index Ultra-low dose, spirometry-controlled CT scanning Sweat Chloride Nasal Lavage (inflammatory markers and microbiome) Fraction of Exhaled Nitric Oxide (FeNO) Liver Ultrasound Liver examination (signs of liver disease) Sputum Collection (inflammatory markers and microbiome) Stool Collection (inflammation, microbiome, fecal elastase) Abdominal symptom questionnaire CFQ-R (quality of life) Adherence to treatment Height, weight, BMI Forced Expiratory volume in 1 second (FEV1) Microbiological culture of airway specimens (clinical laboratories at sites)

The Lead Investigator is Paul McNally, with Prof. Jane Davies as Co-Lead Investigator. The study will operate in collaboration with our academic and clinical partners and the CF registries in Ireland and the UK. The study is supported by the European CF Society Clinical Trials Network (ECFS-CTN). The study is being run as a CTIMP in the UK clinical sites, as determined by the MHRA. In the Irish sites, the HPRA has determined this study to be an observational research study.

Details
Condition Cystic Fibrosis, Cystic Fibrosis Liver Disease, Cystic Fibrosis in Children, Adherence, Medication, Cystic Fibrosis Gastrointestinal Disease
Treatment Kaftrio
Clinical Study IdentifierNCT04602468
SponsorRoyal College of Surgeons, Ireland
Last Modified on31 May 2022

Eligibility

Yes No Not Sure

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
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
patients/parents concerned
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
contraception requirements as detailed in the local Kaftrio SmPC and in line with the
standard of care
Male and female participants of childbearing potential must agree to adhere to

Exclusion Criteria

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
Patients not willing to comply with study procedures or assessments
Individuals on clinical trials of investigational CFTR modulators
Any contraindication to Katrio treatment as per the local approved SmPC
Severe hepatic impairment
Pregnant and breastfeeding women
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