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FARD (RaDiCo Cohort) (RaDiCo-FARD) (FARD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05954416
Recruitment Status : Recruiting
First Posted : July 20, 2023
Last Update Posted : July 20, 2023
Sponsor:
Information provided by (Responsible Party):
Institut National de la Santé Et de la Recherche Médicale, France

Study Description
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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.

Condition or disease
Inherited Epidermolysis Bullosa Ichthyosis Ectodermal Dysplasia Incontinentia Pigmenti Neurofibromatosis Type 1 Albinism Pemphigus Mucous Membrane Pemphigoid Palmoplantar Keratoderma

Study Design
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Study Type : Observational
Estimated 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 : March 7, 2018
Estimated Primary Completion Date : March 7, 2027
Estimated Study Completion Date : March 7, 2027

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Skin Conditions

Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Individual burden score for each selected rare disease [ Time Frame: 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 :
  1. Description of calculated scores based on widely used survey completed by patients [ Time Frame: Through study completion, an average of 5 years ]
  2. Description of calculated scores based on widely used survey completed by parents [ Time Frame: Through study completion, an average of 5 years ]
  3. Description of variations of quality-of-life scores. [ Time Frame: Through study completion, an average of 5 years ]
  4. 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 ]
  5. Descriptive analysis of the socio-economic Burden. [ Time Frame: Through study completion, an average of 5 years ]
  6. Descriptive analysis of the Individual Health Care Cost. [ Time Frame: Through study completion, an average of 5 years ]
  7. 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
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This study concerns patients affected by one the 9 following rare skin diseases: Inherited epidermolysis bullosa, ichthyosis, ectodermal dysplasia, Incontinentia Pigmenti, neurofibromatosis type 1, albinism, pemphigus, mucous membrane pemphigoid, and palmoplantar keratoderma recruited and followed in a reference/competence centre of the healthcare network of rare dermatologic diseases, FIMARAD.
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05954416


Contacts
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Contact: Christine BODEMER + 33 1 44 49 46 72 christine.bodemer@aphp.fr

Locations
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France
Hôpital Avicenne Not yet recruiting
Bobigny, France
Contact: Catherine PROST         
Hôpital des Enfants - Groupe Hospitalier Pellegrin Not yet recruiting
Bordeaux, France
Contact: Christine LEAUTE-LABREZE         
Hôpital des Enfants - Groupe Hospitalier Pellegrin Not yet recruiting
Bordeaux, France
Contact: Marie-Sylvie DOUTRE         
Hôpital Henri-Mondor Not yet recruiting
Créteil, France
Contact: Pierre WOLKENSTEIN         
Hôpital François Mitterrand Not yet recruiting
Dijon, France
Contact: Pierre VABRE         
Hôpital Dupuytren Not yet recruiting
Limoges, France
Contact: Christophe BEDANE         
Hôpital de la Timone Not yet recruiting
Marseille, France
Contact: Marie-Aleth RICHARD         
Hôpital Saint-Eloi Not yet recruiting
Montpellier, France
Contact: Didier BESSIS         
Hôpital l'Archet Recruiting
Nice, France
Contact: Christine CHIAVERINI         
Hôpital Necker-Enfants Malades Recruiting
Paris, France
Contact: Christine BODEMER         
Hôpital Saint-Louis Recruiting
Paris, France
Contact: Emmanuelle BOURRAT         
Hôpital Robert-Debré Not yet recruiting
Reims, France
Contact: Philippe BERNARD         
Hôpital Charles Nicolle Recruiting
Rouen, France
Contact: Pascal JOLY         
Hôpital Larrey Recruiting
Toulouse, France
Contact: Juliette MAZEREEUW-HAUTIER         
Hôpital Trousseau Recruiting
Tours, France
Contact: Annabel MARUANI         
Sponsors and Collaborators
Institut National de la Santé Et de la Recherche Médicale, France
Investigators
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Principal Investigator: Christine BODEMER INSERM UMR 1163
More Information
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Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
ClinicalTrials.gov Identifier: NCT05954416    
Other Study ID Numbers: C16-78
First Posted: July 20, 2023    Key Record Dates
Last Update Posted: July 20, 2023
Last Verified: June 2023

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pemphigoid, Benign Mucous Membrane
Pemphigoid, Bullous
Neurofibromatoses
Neurofibromatosis 1
Albinism
Epidermolysis Bullosa
Ichthyosis
Ectodermal Dysplasia
Keratoderma, Palmoplantar
Incontinentia Pigmenti
Pemphigus
Neurofibroma
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
 Neoplasms
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Skin Diseases
Skin Diseases, Vesiculobullous
Autoimmune Diseases