Cystic FibrosisHORIZONNCT06599892
CF Organization of Care in the Era of Highly Effective Modulator.
An observational study of Cystic Fibrosis, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Recently, triple therapy has made it possible to extend access to these modulators to a large proportion of the patient population, with spectacular clinical benefits, provided the patient carries at least one F508del mutation. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Intervention mapping
- How many people
- 5,000 participants
- Where
- 1 siteFrance
- Study dates
- 1 Sep 2023to 30 Jun 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: In each phase we will use the sample size recommendations for each methodological approach * National surveys by CRCM questionnaire: all national CRCM centres will be contacted by the network. * National registry indicator study = 7513 patients in total, including 5000 eligible for highly effective CFTRm, for the study of pathways at national level, for which clinical and healthcare consumption data are available in the national registry linked to the SNDS. * Case study: 8 to 10 CRCMs, 7 to 8 professionals and 7 to 8 patients per CRCM, i.e. 56 to 80 professionals, 56 to 80 patients.
Who may be able to join
- Patients aged 6 and over with cystic fibrosis treated with CFTRmHE for at least 12 months
- Patients aged over 6 years with cystic fibrosis not eligible for CFTRmHE treatment
- Families of patients under 18 years of age
- Professionals working in a CRCM for more than 12 months, physiotherapists and community nurses involved in the management of cystic fibrosis.
Who cannot join
- Refusal to participate
- Persons deprived of their liberty by judicial or administrative decision
- Persons under psychiatric care
- Adults under legal protection (guardianship, curatorship)
- Persons not affiliated to a social security scheme or beneficiaries of a similar scheme
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Groupement Hospitalier Est Pediatric CFcenter
Lyon
Philippe REIX, MD PhD, 04 27 85 50 42, philippe.reix@chu-lyon.fr
About this study
Official title: Rethinking CF Organization of Care in the Era of Highly Effective Modulator: a Nationwide Research Program HORIZON.
Recently, triple therapy has made it possible to extend access to these modulators to a large proportion of the patient population, with spectacular clinical benefits, provided the patient carries at least one F508del mutation.
The hypothesis is that the arrival of CFTRmHEs has considerably altered the way cystic fibrosis is managed, requiring a rethink of how the CRCMs are organised, and a redefinition of the practices and missions of professionals, as well as the patient-professional relationship.
Read the detailed description
The organisation of cystic fibrosis care in France, within the Muco-CFTR rare disease network, is based on 47 cystic fibrosis resource and competence centres (CRCMs), which bring together multidisciplinary resources (doctors, physiotherapists, nurses, psychologists, dieticians, social workers) to ensure the continuity and coordination of patients' care pathways, taking into account the physical, psychological, educational/professional and social consequences of the disease. The CRCMs' missions are based on the National Diagnosis and Care Protocol (PNDS 2017). The recent arrival of highly effective CFTR modulators (CFTRmHE) in the arsenal of treatments is a therapeutic revolution for 83% of patients in the French Cystic Fibrosis Registry who are eligible for this treatment. Recently, triple therapy has made it possible to extend access to these modulators to a large proportion of the patient population, with spectacular clinical benefits, provided the patient carries at least one F508del mutation. This treatment improves lung function and body mass index, and reduces pulmonary exacerbations, with good tolerability. These results have led to its approval in 2019 in the United States and 2020 in Europe. Real-life results from 245 patients confirmed a spectacular improvement in respiratory function and nutritional status, to the extent that the indication for lung transplantation was suspended in 47 of the 53 patients concerned. The majority of eligible patients started treatment in 2021. The clinical benefits have prompted some patients to abandon standard treatments (respiratory physiotherapy, bronchial fluidisers) and to reconsider the way they are managed at the CRCM (spacing of visits, teleconsultation). However, in the absence of hindsight, it is not possible at present to make recommendations on reducing the burden of care.
The hypothesis is that the arrival of CFTRmHEs has considerably altered the way cystic fibrosis is managed, requiring a rethink of how the CRCMs are organised, and a redefinition of the practices and missions of professionals, as well as the patient-professional relationship.
- Conditions
- Cystic Fibrosis
- Being tested
- Intervention mapping
- Keywords
- Cystic, organisation, relationship, intervention, healthcare, mapping, fibrosis
- Medical subject headings
- Cystic Fibrosis, Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intervention mappingOther
Intervention mapping is a model combining patient and professional questionnaires and a qualitative survey using individual interviews with patients and focus groups with professionals to identify the obstacles and levers to change at the level of patients, professionals and organisations.
| Study group | Role | What they receive |
|---|---|---|
| Patients from the 47 CF French center | National registry indicator study = 7513 patients in total, including 5000 eligible for highly effective CFTRm, for the study of pathways at national level, for which clinical and healthcare consumption data are available in the national registry linked to the SNDS Receives: Other: Intervention mapping | |
| Healthcare professionels | Case study: 8 to 10 CRCMs, 7 to 8 professionals and 7 to 8 patients per CRCM, i.e. 56 to 80 professionals, 200 to 400 patients DELPHI: panel of 80 professionals and patients/parents Receives: Other: Intervention mapping |
What is being measured
The primary outcome is the main question the study is designed to answer.
Obtaining a national consensus on a proposal for a new organisation of the cystic fibrosis care pathway, including the CRCMs and the liberal network
It will be measured by a mixed approach based on the intervention mapping model combining patient and professional questionnaires and a qualitative survey using individual interviews with patients and focus groups with professionals to identify the obstacles and levers to change at the level of patients, professionals and organisations.
The main outcome measure is to establish a new organization of care for pwCF and HCPs in the era of HEMT by the develop an evaluation plan using a community-based participatory approach to promote external validity.
Measured: The outcome measure will be measured at the twelfth month of the survey.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start1 Sep 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion30 Jun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 5,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Philippe REIX, Professor, Principal investigator, Hospices Civils de Lyon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06599892
- Sponsor's protocol ID
- 69HCL23_0749
- Acronym
- HORIZON
- Status last verified
- Sep 2024
- First submitted
- 12 Aug 2024
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rethinking CF Organization of Care in the Era of Highly Effective Modulator: a Nationwide Research Program HORIZON.
- ClinicalTrials.gov ID
- NCT06599892
- Acronym / sponsor ID
- HORIZON / 69HCL23_0749
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic, organisation, relationship, intervention, healthcare, mapping, fibrosis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5,000 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 1 Sep 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 30 Jun 2027 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Recently, triple therapy has made it possible to extend access to these modulators to a large proportion of the patient population, with spectacular clinical benefits, provided the patient carries at least one F508del mutation.
The hypothesis is that the arrival of CFTRmHEs has considerably altered the way cystic fibrosis is managed, requiring a rethink of how the CRCMs are organised, and a redefinition of the practices and missions of professionals, as well as the patient-professional relationship.
Detailed description
The organisation of cystic fibrosis care in France, within the Muco-CFTR rare disease network, is based on 47 cystic fibrosis resource and competence centres (CRCMs), which bring together multidisciplinary resources (doctors, physiotherapists, nurses, psychologists, dieticians, social workers) to ensure the continuity and coordination of patients' care pathways, taking into account the physical, psychological, educational/professional and social consequences of the disease. The CRCMs' missions are based on the National Diagnosis and Care Protocol (PNDS 2017). The recent arrival of highly effective CFTR modulators (CFTRmHE) in the arsenal of treatments is a therapeutic revolution for 83% of patients in the French Cystic Fibrosis Registry who are eligible for this treatment. Recently, triple therapy has made it possible to extend access to these modulators to a large proportion of the patient population, with spectacular clinical benefits, provided the patient carries at least one F508del mutation. This treatment improves lung function and body mass index, and reduces pulmonary exacerbations, with good tolerability. These results have led to its approval in 2019 in the United States and 2020 in Europe. Real-life results from 245 patients confirmed a spectacular improvement in respiratory function and nutritional status, to the extent that the indication for lung transplantation was suspended in 47 of the 53 patients concerned. The majority of eligible patients started treatment in 2021. The clinical benefits have prompted some patients to abandon standard treatments (respiratory physiotherapy, bronchial fluidisers) and to reconsider the way they are managed at the CRCM (spacing of visits, teleconsultation). However, in the absence of hindsight, it is not possible at present to make recommendations on reducing the burden of care.
The hypothesis is that the arrival of CFTRmHEs has considerably altered the way cystic fibrosis is managed, requiring a rethink of how the CRCMs are organised, and a redefinition of the practices and missions of professionals, as well as the patient-professional relationship.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients from the 47 CF French center | – | Other: Intervention mappingNational registry indicator study = 7513 patients in total, including 5000 eligible for highly effective CFTRm, for the study of pathways at national level, for which clinical and healthcare consumption data are available in the national registry linked to the SNDS |
| Healthcare professionels | – | Other: Intervention mappingCase study: 8 to 10 CRCMs, 7 to 8 professionals and 7 to 8 patients per CRCM, i.e. 56 to 80 professionals, 200 to 400 patients DELPHI: panel of 80 professionals and patients/parents |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- In each phase we will use the sample size recommendations for each methodological approach * National surveys by CRCM questionnaire: all national CRCM centres will be contacted by the network. * National registry indicator study = 7513 patients in total, including 5000 eligible for highly effective CFTRm, for the study of pathways at national level, for which clinical and healthcare consumption data are available in the national registry linked to the SNDS. * Case study: 8 to 10 CRCMs, 7 to 8 professionals and 7 to 8 patients per CRCM, i.e. 56 to 80 professionals, 56 to 80 patients.
Key inclusion criteria (4 listed)
- Patients aged 6 and over with cystic fibrosis treated with CFTRmHE for at least 12 months
- Patients aged over 6 years with cystic fibrosis not eligible for CFTRmHE treatment
- Families of patients under 18 years of age
- Professionals working in a CRCM for more than 12 months, physiotherapists and community nurses involved in the management of cystic fibrosis.
Key exclusion criteria (5 listed)
- Refusal to participate
- Persons deprived of their liberty by judicial or administrative decision
- Persons under psychiatric care
- Adults under legal protection (guardianship, curatorship)
- Persons not affiliated to a social security scheme or beneficiaries of a similar scheme
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intervention mappingIntervention mapping is a model combining patient and professional questionnaires and a qualitative survey using individual interviews with patients and focus groups with professionals to identify the obstacles and levers to change at the level of patients, professionals and organisations. | Other | Healthcare professionels, Patients from the 47 CF French center |
Primary outcome
Obtaining a national consensus on a proposal for a new organisation of the cystic fibrosis care pathway, including the CRCMs and the liberal network
Time frame: The outcome measure will be measured at the twelfth month of the survey.
It will be measured by a mixed approach based on the intervention mapping model combining patient and professional questionnaires and a qualitative survey using individual interviews with patients and focus groups with professionals to identify the obstacles and levers to change at the level of patients, professionals and organisations. The main outcome measure is to establish a new organization of care for pwCF and HCPs in the era of HEMT by the develop an evaluation plan using a community-based participatory approach to promote external validity.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Philippe REIX, Professor principal investigator
Hospices Civils de Lyon
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by Hospices Civils de Lyon at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Philippe REIX, Professor (04 27 85 50 42 ext. 04 27 85 67 72, philippe.reix@chu-lyon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Obtaining a national consensus on a proposal for a new organisation of the cystic fibrosis care pathway, including the CRCMs and the liberal network (time frame: The outcome measure will be measured at the twelfth month of the survey.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.