Ignite Creation Date:
2024-10-26 @ 3:40 PM
Last Modification Date:
2024-10-26 @ 3:40 PM
Study NCT ID:
NCT06599892
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-12
Brief Title:
CF Organization of Care in the Era of Highly Effective Modulator
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Rethinking CF Organization of Care in the Era of Highly Effective Modulator a Nationwide Research Program HORIZON
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HORIZON
Brief 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
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
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 educationalprofessional 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
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None