FARD (RaDiCo Cohort) (RaDiCo-FARD)

Официальное название

National Cohort for Evaluation of the Burden of Rare Skin Diseases

Обзор

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.

Первичные конечные точки

• Individual burden score for each selected rare disease [Срок оценки: Through study completion, an average of 5 years]

Вторичные конечные точки (7)

• Description of calculated scores based on widely used survey completed by patients [Срок оценки: Through study completion, an average of 5 years]
• Description of calculated scores based on widely used survey completed by parents [Срок оценки: Through study completion, an average of 5 years]
• Description of variations of quality-of-life scores. [Срок оценки: 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. [Срок оценки: Through study completion, an average of 5 years]
• Descriptive analysis of the socio-economic Burden. [Срок оценки: Through study completion, an average of 5 years]
• Descriptive analysis of the Individual Health Care Cost. [Срок оценки: Through study completion, an average of 5 years]
• Search for association between individual burden score and clinical severity of the disease. [Срок оценки: Through study completion, an average of 5 years]

Критерии участия

Критерии включения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Франция · 15 центров

• 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
• … и ещё 7 центров

Идентификаторы

NCT: NCT05954416 · C16-78

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗