Real world health outcomes in people with cystic fibrosis after initiation of a new combination treatment
| ISRCTN | ISRCTN53454974 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN53454974 |
| EudraCT/CTIS number | 2021-000922-85 |
| IRAS number | 279116 |
| ClinicalTrials.gov number | NCT04602468 |
| Secondary identifying numbers | IRAS 279116, CFF OOC – 2019 |
- Submission date
- 22/10/2020
- Registration date
- 16/12/2020
- Last edited
- 05/04/2024
- Recruitment status
- No longer recruiting
- Overall study status
- Ongoing
- Condition category
- Nutritional, Metabolic, Endocrine
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Plain English Summary
Background and study aims
Cystic fibrosis is an inherited condition that causes sticky mucus to build up in the lungs and digestive system. This causes lung infections and problems with digesting food.
In recent years, a number of new exciting medications have been developed that treat the underlying genetic defect in Cystic fibrosis (CF).
In August 2020 the next generation of potent CF medications Elexacaftor/Tezacaftor/Ivacaftor (ETI) was approved for patients with CF in Europe (having previously been approved by the FDA. This presents a unique opportunity for us to plan a real world study involving all Irish centres in people six years and above with CF.
Who can participate?
People with cystic fibrosis that are suitable for treatment with triple combination modulator therapy can partake in this study.
What does the study involve?
The study will examine clinical outcomes over a seven-year period for each age cohort (6-11 years and 12+ years) across eight pediatric and adult CF centers in Ireland and the UK. Participants will be prescribed ETI. They will attend appointments to provide measurements at the start of the study and then every 3 months for the first 2 year and then annually until the end of the study.
What are the possible benefits and risks of participating?
The subject may benefit from the information obtained during the study. Depending on the usual practice of performing tests and investigations at your local centre, the subject may have more diagnostic tests performed that can be used by the local team. People in research studies see their CF team more than those not involved in research studies. This could be associated with better outcomes.
Almost all medical investigations and treatments have some risks. The following tests are associated with mild discomfort: Nasal lavage (irritating, like doing a nasal rinse). Sputum induction – subject is encouraged to cough after having nebulised hypertonic saline (can cause excessive coughing). Blood tests (we will endeavour to collect blood for RECOVER only when it is already needed for clinical care - annually).
The subject may have CT scans as part of the advanced tests required for this study. They would not have these scans if they did not take part in the study. These procedures use ionising radiation to form images of the lungs. Ionising radiation can cause cell damage that may, after many years or decades, turn cancerous. We are all at risk of developing cancer during our lifetime. This will happen to about 50% of people at some point in their life. Taking part in this study will increase the chances of this happening from 50% to 50.1 %.
Where is the study run from?
RECOVER will be co-ordinated through the Royal College of Surgeons in Ireland.
When is the study starting and how long is it expected to run for?
January 2020 to June 2029
Who is funding the study?
1. Cystic Fibrosis Foundation (USA)
2. Cystic Fibrosis Trust (UK)
3. Cystic Fibrosis Ireland
Who is the main contact?
RECOVER study team, recover@rcsi.ie
RECOVER project manager, Rachel Cregan rachelcregan@rcsi.ie
Contact information
Ms Rachel Cregan
Public
Public
National Children’s Research Centre
Gate 5
Children’s Health Ireland at Crumlin
Dublin
D12 N512
Ireland
| Phone | +353 (0)86 195 3073 |
|---|---|
| rachelcregan@rcsi.ie |
Prof Jane Davies
Scientific
Scientific
Imperial College London and Royal Brompton and Harefield NHS Foundation Trust
Sydney St.
Chelsea
London
SW3 6NP
United Kingdom
 ORCID ID |
0000-0003-3506-1199 |
|---|---|
| Phone | +44 (0)20 7594 7973 |
| j.c.davies@imperial.ac.uk |
Prof Paul McNally
Scientific
Scientific
Royal College of Surgeons in Ireland
Children's Health Ireland at Crumlin
Cooley Road
Dublin
D12 N512
Ireland
| ORCID ID |
0000-0001-7102-1712 |
|---|---|
| Phone | +353 (1)4096500 |
| paulmcnally@rcsi.ie |
Study information
| Study design | Multicenter clinical trial of an investigational medicinal product in UK clinical sites, multicenter observational cohort study in Irish sites |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Non randomised study |
| Study setting(s) | Hospital |
| Study type | Other |
| Participant information sheet | Not available in web format, please use contact details to request a participant information sheet |
| Scientific title | Real world clinical outcomes with novel modifier therapy combinations in people with cystic fibrosis |
| Study acronym | RECOVER |
| Study hypothesis | Current study hypothesis as of 20/03/2024: 1. Use of Elexacaftor/Tezacaftor/Ivacaftor (ETI) in routine clinical practice is associated with significant and sustained improvements in airway and gastrointestinal outcomes and quality of life in children and adults with cystic fibrosis 2. Adherence to routine therapies will decrease after initiation of Elexacaftor/Tezacaftor/Ivacaftor (ETI) _____ Previous study hypothesis: 1. Use of TCMT in routine clinical practice is associated with significant and sustained improvements in airway and gastrointestinal outcomes and quality of life in children and adults with cystic fibrosis 2. Adherence to routine therapies will decrease after initiation of TCMT |
| Ethics approval(s) | 1. Approved 22/03/2021, London - City & East Research Ethics Committee (Bristol Research Ethics Committee Centre, Whitefriars, Level 3, Block B, Lewins Mead, Bristol, BS1 2NT, UK; +44 (0)2071048033/53; cityandeast.rec@hra.nhs.uk ), REC ref: 21/LO/0224 2. Approved 11/03/2020, Children's Health Ireland at Crumlin Medical Research Committee (Crumlin, Dublin 12, Ireland; +353 (0) 14096243; no email provided), ref: Gen/807/20 3. Approved 30/06/2020, Children's Health Ireland at Temple Street ethics committee (Research Office, Temple Street Children’s University Hospital, Temple Street, Dublin 1, Ireland; +353 (0)1 892 1787, research@cuh.ie), ref: 20.020 4. Approved 17/08/2020, Children's Health Ireland at SJH/TUH Research Ethics Committee (Tallaght University Hospital, Dublin 24, Ireland; +353 (0)1-414 2199; researchethics@tuh.ie), ref: 2020-07 5. Approved 11/06/2020, University Hospital Limerick ethics (Research Ethics Committee, UL Hospitals Group, USE, Unit 2, Loughmore Avenue, Raheen Business Park, Limerick, V94P7X9, Ireland; +353 (0)61 482519; joanne.oconnor@hse.ie), ref: 055/2020 6. Approved 27/08/2020, St Vincent’s Healthcare Group Ethics and Medical Research Committee (Education and Research Centre, Elm Park, Dublin 4, Ireland; +353 (0)1-2214117; leona.malone@ucd.ie), ref: RS20-047 |
| Condition | Cystic fibrosis |
| Intervention | Current interventions as of 20/03/2024: Our aim with RECOVER is to examine the clinical impact of ETI 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 in RECOVER to gain important insight into how ETI works, and what impact it has on rescue of CFTR function in this group of people. 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 classified by the regulatory authority, the MHRA. The Irish regulatory authority, the HPRA, has determined this study to be an observational research study and will be run as one at the Irish sites. Subject participation is 7 years. Prior to any study assessments being complete, the participant will be contacted by a member of the study team provided information on the study in age-appropriate information leaflets, and request the participant to review. If the participant is happy to take part in the study, a team member will arrange a day for the participant to come to the clinical study site where the investigator will take informed consent. Once consent has been obtained, the participant have their eligibility assessed. In the UK, this will include a pregnancy test for women of child bearing potential prior to any other study assessments. The subject will be enrolled if they meet all inclusion criteria and no exclusion criteria. They will then undergo several tests at -3 month visit and baseline prior to starting on commercially available ETI (at the dosage decided by their clinician). Once the participant has started this triple combination modulator therapy, they will be monitored at 3 monthly intervals for the first 2 years and then annually after this. _____ Previous interventions as of 29/04/2021: 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 in RECOVER to gain important insight into how Kaftrio works, and what impact it has on rescue of CFTR function in this group of people. 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 classified by the regulatory authority, the MHRA. The Irish regulatory authority, the HPRA, has determined this study to be an observational research study and will be run as one at the Irish sites. Subject participation is 27 months. Prior to any study assessments being complete, the participant will be contacted by a member of the study team provided information on the study in age-appropriate information leaflets, and request the participant to review. If the participant is happy to take part in the study, a team member will arrange a day for the participant to come to the clinical study site where the investigator will take informed consent. Once consent has been obtained, the participant have their eligibility assessed. In the UK, this will include a pregnancy test for women of child bearing potential prior to any other study assessments. The subject will be enrolled if they meet all inclusion criteria and no exclusion criteria. They will then undergo several tests at -3 month visit and baseline prior to starting on commercially available Kaftrio (at the dosage decided by their clinician). Once the participant has started this triple combination modulator therapy, they will be monitored at 3 monthly intervals, which will coincide with the patients 3 monthly clinic visits. _____ Previous interventions: 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 in RECOVER to gain important insight into how Kaftrio works, and what impact it has on rescue of CFTR function in this group of people. 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). Subject participation is 27 months. Prior to any study assessments being complete, the participant will be contacted by a member of the study team provided information on the study in age-appropriate information leaflets, and request the participant to review. If the participant is happy to take part in the study, a team member will arrange a day for the participant to come to the clinical study site where the investigator will take informed consent. Once consent has been obtained, the participant will undergo several tests at -3 month visit and baseline prior to starting on commercially available Kaftrio (at the dosage decided by their clinician). Once the participant has started this triple combination modulator therapy, they will be monitored at 3 monthly intervals, which will coincide with the patients 3 monthly clinic visits. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Elexacaftor/Tezacaftor/Ivacaftor (ETI) |
| Primary outcome measure | Current primary outcome measure as of 20/03/2024: 1. Lung clearance index (LCI or multiple breath washout) is measured to determine the effect of treatment on pulmonary function. It will be measured at -3 months, baseline, 6 months, 12 months, 18months and annually until the end of the study. 2. Spirometry controlled CT will assess the effect of Elexacaftor/Tezacaftor/Ivacaftor (ETI) on the CT scores. It is for patients on the advanced testing group only. It will be measured at baseline, 12 months and 24 months and at years four and six at sites doing routine clinical CT. Scan at year six for sites not performing biennial CT. 3. Height/weight/BMI will be used for pulmonary function and to determine the effects of nutrition. It is measured at -3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and annually until the end of the study. 4. FEV1 (spirometry) is measured to determine the effect of treatment on pulmonary function. It is measured at -3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and annually until the end of the study. 5. Airway sampling (microbiology) will be used the measure the effect on ETI on airway infection and inflammation in children and adults with CF. It will be measured at -3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and annually until the end of the study. 6. Nasal lavage will be used to measure the effect of TCTM on airway infection and inflammation. It is measured on patients in the advanced testing arm only. This will be measured at baseline, 6 months and annually until the end of the study. 7. FeNO (exhaled nitric oxide) is used to measure airway infection and inflammation. It is measured at -3 months, baseline, 6 months and 12 months 8. Liver function testing will be used to determine the effect of ETI on nutrition, gastrointestinal symptoms, gut inflammation and pancreatic function in children and adults over a 2-year period. It is measured at baseline, 3 months, 6 months and annually until the end of the study. 9. Liver ultrasound will be used to determine ETI on nutrition, gastrointestinal symptoms, gut inflammation and pancreatic function. It will be measured annually until the end of the study. 10. Sputum sample collection is for the advanced testing group only. This will be used to determine ETI effect on airway infection and inflammation. It will be measured at baseline, 6 months and annually until the end of the study. 11. Liver examination will be performed annually until the end of the study as part of the nutritional, gastrointestinal, gut inflammation and pancreatic function aim 12. Effect of ETI on digestive tract by stool sample collection will be performed at baseline, 1 month, 6 months and 24 months and annually until the end of the study. 13. Blood sample collection will occur annually until the end of the study. The EDTA blood sample will be used for genetic modifiers of treatment response by our collaborators in Toronto 14. Abdominal symptom questionnaire and score will be used for nutritional and gastrointestinal symptoms outcomes. It is measured at -3 months, baseline, 1 month, 2 months, 6 months and annually until the end of the study. 15. CFQ-R questionnaire will be administered at baseline, 6 months and and annually until the end of the study. This has components on digestion, respiratory and quality of life 16. Pharmacy records (medication pick up rates) will be reviewed at baseline, 12 months and 24 months to assess the impact of the introduction of ETI on antibiotic treatment of pulmonary disease and on adherence 17. Adherence and barriers to adherence questionnaires will be collected at - 3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and 24 months. This will assess adherence impact with overall medical treatments for CF. 18. MEMs caps reading will be at 12 months and will assess adherence to ETI 19. Antibiotic use (through prescribed treatment plan and pharmacy pick up rates) will be measured at baseline, 12 months and 24 months, and will assess the impact of the introduction of ETI on antibiotic treatment for pulmonary disease 20. Effect of ETI on sweat, sweat chloride will be measured at baseline, 6 months and annually until the end of the study. 21. Mental Health Questionnaires will be carried out at year 3 and annually until the end of the study. This is an aim for the five year extension study only. _____ Previous primary outcome measure: 1. Lung clearance index (LCI or multiple breath washout) is measured to determine the effect of treatment on pulmonary function. It will be measured at -3 months, baseline, 6 months, 12 months, 18 months and 24 months 2. Spirometry controlled CT will assess the effect of triple combination modulator therapy (TCMT) on the CT scores. It is for patients on the advanced testing group only. It will be measured at baseline, 12 months and 24 months 3. Height/weight/BMI will be used for pulmonary function and to determine the effects of nutrition. It is measured at -3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and 24 months 4. FEV1 (spirometry) is measured to determine the effect of treatment on pulmonary function. It is measured at -3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and 24 months 5. Airway sampling (microbiology) will be used the measure the effect on TCMT on airway infection and inflammation in children and adults with CF. It will be measured at -3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and 24 months 6. Nasal lavage will be used to measure the effect of TCTM on airway infection and inflammation. It is measured on patients in the advanced testing arm only. This will be measured at baseline, 6 months and 12 months 7. FeNO (exhaled nitric oxide) is used to measure airway infection and inflammation. It is measured at -3 months, baseline, 6 months and 12 months 8. Liver function testing will be used to determine the effect of TCMT on nutrition, gastrointestinal symptoms, gut inflammation and pancreatic function in children and adults over a 2-year period. It is measured at baseline, 3 months, 6 months, 12 months and 24 months 9. Liver ultrasound will be used to determine TCMT on nutrition, gastrointestinal symptoms, gut inflammation and pancreatic function. It will be measured at baseline, 12 months and 24 months 10. Sputum sample collection is for the advanced testing group only. This will be used to determine TCMT effect on airway infection and inflammation. It will be measured at baseline, 6 months, 12 months and 24 months 11. Liver examination will be performed at baseline, 12 months and 24 months as part of the nutritional, gastrointestinal, gut inflammation and pancreatic function aim 12. Effect of TCMT on digestive tract by stool sample collection will be performed at baseline, 1 month, 6 months and 24 months as above 13. Blood sample collection will occur at baseline, 12 months and 24 months. The EDTA blood sample will be used for genetic modifiers of treatment response by our collaborators in Toronto 14. Abdominal symptom questionnaire and score will be used for nutritional and gastrointestinal symptoms outcomes. It is measured at -3 months, baseline, 1 month, 2 months, 6 months, 12 months and 24 months 15. CFQ-R questionnaire will be administered at baseline, 6 months, 12 months and 24 months. This has components on digestion, respiratory and quality of life 16. Pharmacy records (medication pick up rates) will be reviewed at baseline, 12 months and 24 months to assess the impact of the introduction of TCMT on antibiotic treatment of pulmonary disease and on adherence 17. Adherence and barriers to adherence questionnaires will be collected at - 3 months, baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 18 months, 21 months and 24 months. This will assess adherence impact with overall medical treatments for CF 18. MEMs caps reading will be at 12 months and will assess adherence to TCMT 19. Antibiotic use (through prescribed treatment plan and pharmacy pick up rates) will be measured at baseline, 12 months and 24 months, and will assess the impact of the introduction of TCMT on antibiotic treatment for pulmonary disease 20. Effect of TCMT on sweat, sweat chloride will be measured at baseline, 3 months, 12 months and 24 months |
| Secondary outcome measures | There are no secondary outcome measures |
| Overall study start date | 01/01/2020 |
| Overall study end date | 30/06/2029 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Mixed |
| Lower age limit | 6 Years |
| Sex | Both |
| Target number of participants | 254 |
| Total final enrolment | 206 |
| Participant inclusion criteria | Inclusion Criteria for Parent Study (UK CTIMP Sites) 1. 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. Inclusion Criteria for Subjects on ETI 1. 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. 2. 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. 3. 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. Inclusion Criteria for Extension Study (UK CTIMP Sites) 1. Children and adults with CF who have completed two years participation on the parent study, and are willing to provide informed consent for continued data and bio-sample collection for a period of five years. _____ Previous inclusion criteria as of 29/04/2021: Inclusion Criteria for RECOVER CTIMP (UK clinical sites only): 1. 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 2. 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. 3. 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. 4. 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). 5. 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. 6. 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. Inclusion Criteria for RECOVER Observational Study (Irish Sites Only): 1. Children and adults with CF independently determined to commence on triple combination CFTR modulator treatment as covered by the license given by the manufacturer. 2. As this is a real-world study, all eligible subjects, including those with FEV1 values lower than 40% or greater than 90% and/or those with significant comorbidity or multi-resistant or atypical organisms will also be included in the study. 3. Subjects must be taking the full dose of the triple combination compound. _____ Previous inclusion criteria: 1. Children and adults with CF and starting on triple combination CFTR modulator treatment 2. Subjects must be taking the full dose of the triple combination compound. |
| Participant exclusion criteria | Current exclusion criteria as of 20/03/2024: Exclusion Criteria for Parent Study (UK CTIMP Sites) 1. Patients not willing to comply with study procedures or assessments. 2. Individuals on clinical trials of investigational CFTR modulators. 3. 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. 4. Any contraindication to ETI treatment as per the local approved SmPC. 5. Severe hepatic impairment. 6. Pregnant and breastfeeding women. Exclusion Criteria for Extension Study (UK CTIMP Sites) 1. Participants not willing to comply with study procedures or assessments. _____ Previous exclusion criteria as of 29/04/2021: Exclusion Criteria for RECOVER CTIMP (UK clinical sites only): 1. Patients not willing to comply with study procedures or assessments 2. Individuals on clinical trials of investigational CFTR modulators 3. 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 4. Any contraindication to Katrio treatment as per the local approved SmPC 5. Severe hepatic impairment 6. Pregnant and breastfeeding women Exclusion Criteria for RECOVER Observational Study (Irish Sites Only): 1. Patients not willing to comply with study procedures or assessments. Individuals on clinical trials of investigational CFTR modulators. 2. 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. _____ Previous exclusion criteria: 1. Patients not willing to comply with study procedures or assessments 2. Individuals on clinical trials of investigational CFTR modulators. |
| Recruitment start date | 01/08/2020 |
| Recruitment end date | 18/05/2023 |
Locations
Countries of recruitment
- England
- Ireland
- Northern Ireland
- United Kingdom
Study participating centres
Children's Health Ireland at Crumlin
Crumlin
Dublin
D12 N512
Ireland
Dublin
D12 N512
Ireland
Children's Health Ireland at Temple Street
Temple Street
Dublin
D01 YC67
Ireland
Dublin
D01 YC67
Ireland
Children's Health Ireland at Tallaght
Tallaght
Dublin
D24 NR0A
Ireland
Dublin
D24 NR0A
Ireland
University Hospital Limerick
Children's Arc
Butterfly ward
St. Nessans Rd
Dooradoyle
Limerick
V94 F858
Grenada
Butterfly ward
St. Nessans Rd
Dooradoyle
Limerick
V94 F858
Grenada
St. Vincent's University Hospital
Merrion Road
Dublin
D04 N2E0
Ireland
Dublin
D04 N2E0
Ireland
Guy's and St Thomas' NHS Foundation Trust
Sydney St
Chelsea
London
SW3 6NP
United Kingdom
Chelsea
London
SW3 6NP
United Kingdom
Royal Belfast Hospital for Sick Children
274 Grosvenor Rd
Belfast
BT12 6BA
United Kingdom
Belfast
BT12 6BA
United Kingdom
Sponsor information
Royal College of Surgeons in Ireland
University/education
University/education
123 St Stephen's Green
Saint Peter's
Dublin
D02 YN77
Ireland
| Phone | +353 (0)1-8093863 |
|---|---|
| sponsorship@rcsi.ie | |
| Website | https://www.rcsi.com/dublin |
"ROR" |
https://ror.org/01hxy9878 |
Funders
Funder type
Charity
Cystic Fibrosis Foundation
Government organisation / Trusts, charities, foundations (both public and private)
Government organisation / Trusts, charities, foundations (both public and private)
- Alternative name(s)
- CF Foundation, CFF
- Location
- United States of America
Cystic Fibrosis Trust
No information available
Cystic Fibrosis Ireland
No information available
Results and Publications
| Intention to publish date | 13/09/2023 |
|---|---|
| Individual participant data (IPD) Intention to share | Yes |
| IPD sharing plan summary | Other |
| Publication and dissemination plan | Dissemination will be a continuous process throughout the project as results are obtained, and dissemination channels reviewed annually to determine if an update is needed as new information comes to light. The Management Group will determine when an update is required. Our objectives are to disseminate results and project achievements to international stakeholders, thought-leaders, and equivalent institutions worldwide through structured dissemination activities. Given that most project results will be clinical/scientific in nature, we anticipate that the key audiences will be clinicians and researchers in industry and academia. These audiences will be reached via publicly accessible scientific publications (peer-reviewed high impact journals) and presentations at international conferences such as the North American Cystic Fibrosis Conference (NACFC) and European CF Conference. SciVal and Altmetrics (https://www.altmetric.com/) will be used to measure research impact, providing evidence of the broad reach and potential future impacts of research. Altmetric data will be reported (using Leiden Manifesto principles) to give an accurate indication of attention and engagement generated by individual outputs, which will be used to improve the reporting and dissemination of our research/. All collaborators will participate in dissemination, and funding for high-quality dissemination, and communications have been justified in the budget template, including publication costs, and conference registration fees. |
| IPD sharing plan | All data generated or analysed during this study will be included in the subsequent results publication. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| HRA research summary | 26/07/2023 | No | No | ||
| Protocol file | version 7.0 | 09/01/2024 | 20/03/2024 | No | No |
| Results article | 01/11/2023 | 20/03/2024 | Yes | No |
Additional files
Editorial Notes
05/04/2024: Contact details updated.
20/03/2024: The following changes were made to the trial record:
1. Uploaded protocol (not peer-reviewed) as an additional file.
2. The study hypothesis was changed.
3. The interventions were changed.
4. The primary outcome measure was changed.
5. The study website was changed from "https://recovercf.ie/"; to"https://www.realworld4cf.com/recover/";.
6. The inclusion criteria were changed.
7. The total final enrolment was added.
8. The exclusion criteria were changed.
9. The recruitment end date was changed from 30/03/2024 to 18/05/2023.
10. The overall end date was changed from 30/06/2024 to 30/06/2029.
11. The plain English summary was updated to reflect these changes.
12. Publication reference added.
13. The drug name was changed from "Kaftrio (ivacaftor, tezacaftor, and elexacaftor)" to "Elexacaftor/Tezacaftor/Ivacaftor (ETI)".
14. The intention to publish date was changed from 30/06/2024 to 13/09/2023.
29/04/2021: The following changes were made to the trial record:
1. Ethics approval details, EudraCT, ClinicalTrials.gov and protocol numbers added.
2. The study design was changed from 'Multicenter observational cohort study' to 'Multicenter clinical trial of an investigational medicinal product in UK clinical sites, multicenter observational cohort study in Irish sites'.
3. The interventions, inclusion and exclusion criteria were updated.
4. The target number of participants was changed from 237 to 254.
5. The recruitment start date was changed from 27/08/2020 to 01/08/2020.
6. The trial participating centres were updated to update Royal Brompton Hospital to Guy's and St Thomas' NHS Foundation Trust, and to remove Belfast City Hospital.
03/11/2020: Trial’s existence confirmed by South West- Cornwall & Plymouth Research Ethics Committee.
