Ignite Creation Date:
2024-10-26 @ 3:42 PM
Last Modification Date:
2024-10-26 @ 3:42 PM
Study NCT ID:
NCT06636344
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-10-02
Brief Title:
Impact of Optimized Recruitment and Follow-up of Patients with Pseudoxanthoma Elasticum PXE
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Impact of Optimized Recruitment and Follow-up of Patients with Pseudoxanthoma Elasticum PXE by the PXE Reference Center At the CHU in Angers France Thanks to the Implementation of Alternating Pathways Adapted to Age and Symptomatology and Including Teleconsultations
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
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:
REMOTE-PXE
Brief Summary:
Pseudoxanthoma elasticum PXE is a rare genetic disorder characterized by ectopic calcifications in the skin retina and arterial walls Angers University Hospital is the national rare disease reference center CRMR for PXE Although PXE is hereditary its main clinical manifestations unsightly skin lesions intermittent arterial claudication stroke retinal bleeding and blindness are delayed and slowly progress over the course of a lifetime They are rarely life-threatening but have a major functional impact To date management of PXE is purely preventive and symptomatic Three successive states can be individualized during PXE course corresponding to three very different patient profiles in terms of age clinical manifestations occurrence of complications and their treatment
PXE is essentially a severe disease in adults in the second half of life This contrasts with the presence of many patients seen for their follow-up at school age or in employment and at the age of children It is therefore necessary to optimize the recruitment of PXE patients and to rethink their follow-up by the CRMR
The investigators hypothesize that the implementation of alternating treatment paths better adapted to each of the three patient profiles including multidisciplinary teleconsultations will not only increase the number of patients monitored by the CRMR and benefit from referral care but also to optimize care for greater patient satisfaction their local doctors and the CRMR team
PXE is essentially a severe disease in adults in the second half of life This contrasts with the presence of many patients seen for their follow-up at school age or in employment and at the age of children It is therefore necessary to optimize the recruitment of PXE patients and to rethink their follow-up by the CRMR
The investigators hypothesize that the implementation of alternating treatment paths better adapted to each of the three patient profiles including multidisciplinary teleconsultations will not only increase the number of patients monitored by the CRMR and benefit from referral care but also to optimize care for greater patient satisfaction their local doctors and the CRMR team
Detailed Description:
This is a quasi-experimental study evaluating the impact of new support modalities according to a beforeafter design with an intermediate transition period of 2 years necessary for the proper deployment of these new modalities The study includes 3 separate periods of 2 years from its implementation
Period A1-2 continuation of the recruitment monitoring and care methods for PXE patients as they are currently carried out This period will allow the collection of baseline evaluation criteria
Period A3-4 intermediate period without evaluationmonitoring of indicators gradual implementation of the internal organizational changes necessary for the deployment of alternating pathways
Period A5-6 second period of interest for the evaluation of indicators evaluation of fully operational alternating pathways for the recruitment monitoring and care of PXE patients This period will allow the collection of evaluation criteria and comparison with those of baseline
For the main objective and the first secondary objectives relating to the optimization of the care pathway the analysis will be carried out using global activity data at the CRMR level non-identifying aggregated data
This research involving humans is qualified as non-interventional research because to respond to
the second secondary objective about Evaluation of the impact of alternating courses adapted to age and clinical symptoms on the following criteria patients will answer questionnaires specific to the study quality of life satisfaction and confidence for the patient and the treating physicians involved
the third secondary objective semi-directed interviews will be carried out for patients relatives except parents and treating physicians who agree to participate in the qualitative sub-study
The overall activity data of the CRMR will be collected retrospectively and prospectively on the basis of all source data in order to characterize the evolution of the active file in terms of patients duly followed up contacts without follow-up or lost to follow-up during periods A1-A2 and A5-A6
Period A1-2 continuation of the recruitment monitoring and care methods for PXE patients as they are currently carried out This period will allow the collection of baseline evaluation criteria
Period A3-4 intermediate period without evaluationmonitoring of indicators gradual implementation of the internal organizational changes necessary for the deployment of alternating pathways
Period A5-6 second period of interest for the evaluation of indicators evaluation of fully operational alternating pathways for the recruitment monitoring and care of PXE patients This period will allow the collection of evaluation criteria and comparison with those of baseline
For the main objective and the first secondary objectives relating to the optimization of the care pathway the analysis will be carried out using global activity data at the CRMR level non-identifying aggregated data
This research involving humans is qualified as non-interventional research because to respond to
the second secondary objective about Evaluation of the impact of alternating courses adapted to age and clinical symptoms on the following criteria patients will answer questionnaires specific to the study quality of life satisfaction and confidence for the patient and the treating physicians involved
the third secondary objective semi-directed interviews will be carried out for patients relatives except parents and treating physicians who agree to participate in the qualitative sub-study
The overall activity data of the CRMR will be collected retrospectively and prospectively on the basis of all source data in order to characterize the evolution of the active file in terms of patients duly followed up contacts without follow-up or lost to follow-up during periods A1-A2 and A5-A6
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