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Register of Patients With Prader-Willi Syndrome

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ClinicalTrials.gov Identifier: NCT02829684
Recruitment Status : Recruiting
First Posted : July 12, 2016
Last Update Posted : January 5, 2018
Sponsor:
Information provided by (Responsible Party):
University Hospital, Toulouse

Brief Summary:

Prader-Willi Syndrome (PWS) is a rare syndrome with a prevalence of 15 to 20 000 at birth. PWS represents a large fraction of mental retardation syndromes due to a genetic cause and the most frequent cause of genetic obesity. The majority of the patients are seen by paediatricians. This syndrome is responsible for severe physical, psychological and social impairments.

The diversity and the severity of the manifestations of this disease explain the requirement of multidisciplinary care which deserve specific evaluation. Today the follow-up and management of a great proportion of these patients are greatly insufficient if not absent.

Teams strongly lack information on the natural history of this severe disease and on the factors involved in its evolution and the outcome of these patients throughout life. The present project is to implement a register in the whole country for children and adult patients


Condition or disease Intervention/treatment
Prader-Willi Syndrome Other: Data collection

Study Type : Observational
Estimated Enrollment : 500 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Implementation of a National Register of Children and Adults Presenting Prader-Willi Syndrome
Actual Study Start Date : March 2009
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine



Intervention Details:
  • Other: Data collection
    This register will follow the evolution of the clinical practices and their consequences in the health of the patients, in all regions of France in order to have a national register. for Children and adults.


Primary Outcome Measures :
  1. collect data about patients [ Time Frame: Baseline ]
    Circumstances of diagnosis, genetic diagnosis, modalities of follow-up and clinical management and a questionnaire to evaluate quality of life of the family and social data


Secondary Outcome Measures :
  1. collect data about patients [ Time Frame: During 10 years at least ]
    modalities of follow-up and clinical management and a questionnaire to evaluate quality of life of the family and social data



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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
Patients will be included by the paediatric endocrinologists or the endocrinologists for adults of each region based on their own recruitment
Criteria

Inclusion Criteria:

  • all subjects with a Prader-Willi Syndrome

Exclusion Criteria:


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): NCT02829684


Contacts
Contact: TAUBER Maité, MD PhD tauber.m@chu-toulouse.fr
Contact: MOLINAS Catherine, CRA molinas.c@chu-toulouse.fr

Locations
France
University Hospital of Children Recruiting
Toulouse, France, 31059
Sponsors and Collaborators
University Hospital, Toulouse
Investigators
Principal Investigator: Tauber Maité, MD University Hospital, Toulouse

Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT02829684     History of Changes
Other Study ID Numbers: 07 315 03
First Posted: July 12, 2016    Key Record Dates
Last Update Posted: January 5, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders