FARD (RaDiCo Cohort) (RaDiCo-FARD)

Sponsor

Institut National de la Santé Et de la Recherche Médicale, France (Other)

Overall Status

Recruiting

CT.gov ID

NCT05954416

Collaborator

(none)

900

Enrollment

Locations

108

Anticipated Duration (Months)

Patients Per Site

0.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The goal of this observational study is to conduct a prospective assessment of the individual Burden of 9 rare skin diseases to assess disability in the broadest sense of the term (psychological, social, economic and physical) for patients and/or families.

Two types of indicators will be used to reach this objective :

an individual burden score calculated based on a burden questionnaire created specifically, approved and designed to understand the tendency to changes in care and lifestyles. The burden questionnaire should be used by patients and/or their family themselves in self-assessment.
a descriptive analysis of all resources (medical and non-medical) used by the family unit to manage the disease.

Condition or Disease	Intervention/Treatment	Phase
Inherited Epidermolysis Bullosa Ichthyosis Ectodermal Dysplasia Incontinentia Pigmenti Neurofibromatosis Type 1 Albinism Pemphigus Mucous Membrane Pemphigoid Palmoplantar Keratoderma

Study Design

Study Type:

Observational

Anticipated Enrollment :

900 participants

Observational Model:

Cohort

Time Perspective:

Other

Official Title:

National Cohort for Evaluation of the Burden of Rare Skin Diseases

Actual Study Start Date :

Mar 7, 2018

Anticipated Primary Completion Date :

Mar 7, 2027

Anticipated Study Completion Date :

Mar 7, 2027

Outcome Measures

Primary Outcome Measures

Individual burden score for each selected rare disease [Through study completion, an average of 5 years]
Before 16 years old, we will focus on the burden of families. After 16 years old, the patient's parent will continue to answer to the family Burden questionnaire and the patient will start to answer to the adult's Burden questionnaire.

Secondary Outcome Measures

Description of calculated scores based on widely used survey completed by patients [Through study completion, an average of 5 years]
Description of calculated scores based on widely used survey completed by parents [Through study completion, an average of 5 years]
Description of variations of quality-of-life scores. [Through study completion, an average of 5 years]
Validation of the clinical severity score for disease which have none at the beginning of the study and description of clinical severity score. [Through study completion, an average of 5 years]
Descriptive analysis of the socio-economic Burden. [Through study completion, an average of 5 years]
Descriptive analysis of the Individual Health Care Cost. [Through study completion, an average of 5 years]
Search for association between individual burden score and clinical severity of the disease. [Through study completion, an average of 5 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion criteria :

adults or children with a confirmed diagnosis of one of the 9 following rare skin disease: Inherited epidermolysis bullosa, Ichthyosis, Ectodermal dysplasia, Incontinetia Pigmenti, Neurofibromatosis type 1, Albinism, Pemphigus, Mucous membrane pemphigoid or Palmoplantar keratoderma.
prevalent or incident and followed in one the reference/competence centers of the FIMARAD healthcare network,
able to understand a survey (for child, survey should be understood by parents),
having given their signed consent to participate to the cohort RaDiCo-FARD (parents' consent for child).

Non-inclusion criteria :

Patients, for whom regular care follow-up is not feasible with the FIMARAD healthcare network sites,
Unconfirmed diagnosis (according to criteria for each disease),
Patients (and/or parents) not able to understand a survey
Patients (and/or parents) not having given their signed consent to participate to the study

Contacts and Locations

Locations

	Site	City	Country
1	Hôpital Avicenne	Bobigny	France
2	Hôpital des Enfants - Groupe Hospitalier Pellegrin	Bordeaux	France
3	Hôpital des Enfants - Groupe Hospitalier Pellegrin	Bordeaux	France
4	Hôpital Henri-Mondor	Créteil	France
5	Hôpital François Mitterrand	Dijon	France
6	Hôpital Dupuytren	Limoges	France
7	Hôpital de la Timone	Marseille	France
8	Hôpital Saint-Eloi	Montpellier	France
9	Hôpital l'Archet	Nice	France
10	Hôpital Necker-Enfants Malades	Paris	France
11	Hôpital Saint-Louis	Paris	France
12	Hôpital Robert-Debré	Reims	France
13	Hôpital Charles Nicolle	Rouen	France
14	Hôpital Larrey	Toulouse	France
15	Hôpital Trousseau	Tours	France