FARD (RaDiCo Cohort) (RaDiCo-FARD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Inherited Epidermolysis Bullosa, Ichthyosis, Ectodermal Dysplasia, Incontinentia Pigmenti. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
National Cohort for Evaluation of the Burden of Rare Skin Diseases
Overview
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.
Primary outcome measures
- Individual burden score for each selected rare disease [Time frame: Through study completion, an average of 5 years]
Secondary outcome measures (7)
- Description of calculated scores based on widely used survey completed by patients [Time frame: Through study completion, an average of 5 years]
- Description of calculated scores based on widely used survey completed by parents [Time frame: Through study completion, an average of 5 years]
- Description of variations of quality-of-life scores. [Time frame: 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. [Time frame: Through study completion, an average of 5 years]
- Descriptive analysis of the socio-economic Burden. [Time frame: Through study completion, an average of 5 years]
- Descriptive analysis of the Individual Health Care Cost. [Time frame: Through study completion, an average of 5 years]
- Search for association between individual burden score and clinical severity of the disease. [Time frame: Through study completion, an average of 5 years]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
France · 15 centers
- Hôpital Avicenne — Bobigny
- Hôpital des Enfants - Groupe Hospitalier Pellegrin — Bordeaux
- Hôpital des Enfants - Groupe Hospitalier Pellegrin — Bordeaux
- Hôpital Henri-Mondor — Créteil
- Hôpital François Mitterrand — Dijon
- Hôpital Dupuytren — Limoges
- Hôpital de la Timone — Marseille
- Hôpital Saint-Eloi — Montpellier
- … and 7 more centers
Identifiers
NCT: NCT05954416 · C16-78