NCT04602468

Brief 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.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
237

participants targeted

Target at P75+ for phase_4

Timeline
38mo left

Started Sep 2020

Longer than P75 for phase_4

Geographic Reach
2 countries

7 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress64%
Sep 2020Jul 2029

Study Start

First participant enrolled

September 3, 2020

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

October 20, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

October 26, 2020

Completed
8.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2029

Expected
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2029

Last Updated

May 2, 2024

Status Verified

May 1, 2024

Enrollment Period

8.6 years

First QC Date

October 20, 2020

Last Update Submit

May 1, 2024

Conditions

Cystic FibrosisCystic Fibrosis Liver DiseaseCystic Fibrosis in ChildrenAdherence, MedicationCystic Fibrosis Gastrointestinal Disease

Outcome Measures

Primary Outcomes (1)

  • To determine the effect of treatment with TCMT on pulmonary function (FEV1 and LCI) in children and adults with CF over a period of 2 years.

    Spirometry, LCI

    24 month period

Secondary Outcomes (5)

  • To determine the effect of treatment with TCMT on spirometry-controlled CT scores in children and adults with CF over a two-year period.

    24 month period

  • To determine the effect of treatment with TCMT on airway infection and inflammation in children and adults with CF

    24 month period

  • To determine the effect of treatment with TCMT on nutrition, gastrointestinal symptoms, gut inflammation and pancreatic function in children and adults with CF over a two-year period

    24 month period

  • To determine the effect of treatment with TCMT on antibiotic treatment of pulmonary disease in children and adults with CF over a two-year period

    24 month period

  • To assess the impact of the introduction of TCMT on adherence with overall medical treatments for CF

    24 month period

Study Arms (2)

Standard group

OTHER

The standard testing group will be available for both age cohorts with sites having a predefined recruitment cap for each testing group. The standard testing will involve the following assessments; sweat chloride, LCI, height/weight/BMI, FEV1, airway sampling (micro), FeNO, liver function testing, liver ultrasound, liver examination, stool collection, blood collection, abdominal symptom score, CFQ-R, pharmacy records medication pick up rate, adherence questionnaires, MEMs caps and antibiotic use.

Drug: Kaftrio

Advanced group

OTHER

In addition to all elements of the standard testing group, the advanced testing group will undergo: Ultra-low dose spirometry-controlled CT scanning, sputum collection and nasal lavage collection. This will be available for both age cohorts with sites having a predefined recruitment cap for each testing group.

Drug: Kaftrio

Interventions

KaftrioDRUG

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.

Also known as: Trikafta
Advanced groupStandard group

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Children's Health Ireland at Crumlin

Dublin, Leinster, Ireland

Location

Children's Health Ireland at Temple Street

Dublin, Ireland

Location

St. Vincent's University Hospital

Dublin, Ireland

Location

University Hospital Limerick

Limerick, V94 F858, Ireland

Location

Children's Health Ireland at Tallaght

Tallaght, Ireland

Location

Royal Belfast Hospital for Sick Children

Belfast, United Kingdom

Location

Royal Brompton Hospital

London, United Kingdom

Location

Related Publications (1)

  • McNally P, Lester K, Stone G, Elnazir B, Williamson M, Cox D, Linnane B, Kirwan L, Rea D, O'Regan P, Semple T, Saunders C, Tiddens HAWM, McKone E, Davies JC; RECOVER Study Group. Improvement in Lung Clearance Index and Chest Computed Tomography Scores with Elexacaftor/Tezacaftor/Ivacaftor Treatment in People with Cystic Fibrosis Aged 12 Years and Older - The RECOVER Trial. Am J Respir Crit Care Med. 2023 Nov 1;208(9):917-929. doi: 10.1164/rccm.202308-1317OC.

    PMID: 37703083DERIVED

Related Links

MeSH Terms

Conditions

Cystic FibrosisMedication Adherence

Interventions

elexacaftor, ivacaftor, tezacaftor drug combination

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesPatient CompliancePatient Acceptance of Health CareTreatment Adherence and ComplianceHealth BehaviorBehavior

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NON RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Masking Details
No blinding. The decision to implement Kaftrio treatment is made completely independently of the decision to enter the study. Those determined to begin treatment on Kaftrio clinically will undergo eligibility assessment.
Purpose
OTHER
Intervention Model
SINGLE GROUP
Model Details: The decision to implement Kaftrio treatment is made completely independently of the decision to enter the study. The investigators will follow the requirements of the local approved Kaftrio SmPC for the patient management as detailed in the 'special warnings and precautions for use' (e.g. management of hepatic impairment, rash, ophthalmological monitoring, between others), and for the management of interaction with other medicinal products and other forms of interactions. The 12+ cohort will be enrolled first on the basis the drug will be approved for children aged 12 and over first. The 6-11 year cohort will only be enrolled when the license is extended to this age group, and treatment with Kaftrio will only occur in the context of prescription by a physician in compliance with marketing authorization and the SPC.
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 20, 2020

First Posted

October 26, 2020

Study Start

September 3, 2020

Primary Completion (Estimated)

April 1, 2029

Study Completion (Estimated)

July 1, 2029

Last Updated

May 2, 2024

Record last verified: 2024-05

Locations

IE(5)GB(2)