FARD (RaDiCo Cohort) (RaDiCo-FARD)

Neurofibromatosis Type 1 Clinical Trial

— FARD  

Official title:

National Cohort for Evaluation of the Burden of Rare Skin Diseases

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05954416
• Other study ID #: C16-78
• Status: Recruiting
• Start date: March 7, 2018
• Est. completion date: March 7, 2027

Study information

• Verified date: June 2023
• Source: Institut National de la Santé Et de la Recherche Médicale, France
• Contact: Christine BODEMER
• Phone: + 33 1 44 49 46 72
• Email: christine.bodemer[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 900
• Est. completion date: March 7, 2027
• Est. primary completion date: March 7, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Albinism
• Ectodermal Dysplasia
• Epidermolysis Bullosa
• Ichthyosis
• Incontinentia Pigmenti
• Inherited Epidermolysis Bullosa
• Mucous Membrane Pemphigoid
• Neurofibromatoses
• Neurofibromatosis 1
• Neurofibromatosis Type 1
• Pemphigoid, Bullous
• Pemphigus

Locations

Country	Name	City	State
France	Hôpital Avicenne	Bobigny
France	Hôpital des Enfants - Groupe Hospitalier Pellegrin	Bordeaux
France	Hôpital des Enfants - Groupe Hospitalier Pellegrin	Bordeaux
France	Hôpital Henri-Mondor	Créteil
France	Hôpital François Mitterrand	Dijon
France	Hôpital Dupuytren	Limoges
France	Hôpital de la Timone	Marseille
France	Hôpital Saint-Eloi	Montpellier
France	Hôpital l'Archet	Nice
France	Hôpital Necker-Enfants Malades	Paris
France	Hôpital Saint-Louis	Paris
France	Hôpital Robert-Debré	Reims
France	Hôpital Charles Nicolle	Rouen
France	Hôpital Larrey	Toulouse
France	Hôpital Trousseau	Tours

Sponsors (1)

Lead Sponsor	Collaborator
Institut National de la Santé Et de la Recherche Médicale, France

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Individual burden score for each selected rare disease	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.	Through study completion, an average of 5 years
Secondary	Description of calculated scores based on widely used survey completed by patients		Through study completion, an average of 5 years
Secondary	Description of calculated scores based on widely used survey completed by parents		Through study completion, an average of 5 years
Secondary	Description of variations of quality-of-life scores.		Through study completion, an average of 5 years
Secondary	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
Secondary	Descriptive analysis of the socio-economic Burden.		Through study completion, an average of 5 years
Secondary	Descriptive analysis of the Individual Health Care Cost.		Through study completion, an average of 5 years
Secondary	Search for association between individual burden score and clinical severity of the disease.		Through study completion, an average of 5 years