FARD (RaDiCo Cohort) (RaDiCo-FARD)

Recruiting

• Conditions: Incontinentia Pigmenti; Albinism; Palmoplantar Keratoderma; Inherited Epidermolysis Bullosa; Ectodermal Dysplasia; Neurofibromatosis Type 1; Pemphigus; Ichthyosis; Mucous Membrane Pemphigoid

• Registration Number: NCT05954416
• Lead Sponsor: Institut National de la Santé Et de la Recherche Médicale, France

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 :

1. 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.

2. a descriptive analysis of all resources (medical and non-medical) used by the family unit to manage the disease.

Detailed Description

Not available

Study Timeline

• Study Start: 2018-03-07
• Status Verified: 2023-06
• Study First Submitted: 2023-06-26
• Study First Submitted QC: 2023-07-18
• Last Update Submitted: 2023-07-18
• Study First Posted: 2023-07-20
• Last Update Posted: 2023-07-20
• Primary Completion: 2027-03-07
• Study Completion: 2027-03-07

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 900

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Description of calculated scores based on widely used survey completed by parents	Through study completion, an average of 5 years
Descriptive analysis of the socio-economic Burden.	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 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
Description of calculated scores based on widely used survey completed by patients	Through study completion, an average of 5 years

Trial Locations

Locations (14)

Hôpital Avicenne; 🇫🇷 Bobigny, France

Hôpital Dupuytren; 🇫🇷 Limoges, France

Hôpital Saint-Eloi; 🇫🇷 Montpellier, France

Hôpital l'Archet; 🇫🇷 Nice, France

Hôpital de la Timone; 🇫🇷 Marseille, France

Hôpital Robert-Debré; 🇫🇷 Reims, France

Hôpital Larrey; 🇫🇷 Toulouse, France

Hôpital Trousseau; 🇫🇷 Tours, France

Hôpital Charles Nicolle; 🇫🇷 Rouen, France

Hôpital des Enfants - Groupe Hospitalier Pellegrin; 🇫🇷 Bordeaux, France

Hôpital Henri-Mondor; 🇫🇷 Créteil, France

Hôpital François Mitterrand; 🇫🇷 Dijon, France

Hôpital Necker-Enfants Malades; 🇫🇷 Paris, France

Hôpital Saint-Louis; 🇫🇷 Paris, France