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Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity (HOGRID)

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ClinicalTrials.gov Identifier: NCT04768803
Recruitment Status : Unknown
Verified February 2021 by University Hospital, Toulouse.
Recruitment status was:  Not yet recruiting
First Posted : February 24, 2021
Last Update Posted : February 24, 2021
Sponsor:
Information provided by (Responsible Party):
University Hospital, Toulouse

Study Description
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Brief Summary:

A significantly higher proportion of patients with rare diseases (RD) with intellectual disability (ID), present hyperphagia, overweight or obesity, compared to the general population. Prader-Willi syndrome is the only genetic obesity identified to date associated with hyperghrelinemia, while ghrelin levels are lower than in controls in other situations of obesity.

The aim of the study is to find out whether the levels of ghrelin, which are abnormally high in PWS throughout life, are also high in these RD when people have hyperphagia and/or overweight.


Condition or disease Intervention/treatment
Angelman Syndrome Smith-Magenis Syndrome X Fragile Syndrome Epilepsy Prader-Willi Syndrome Biological: acylated and unacylated ghrelin dosages

Detailed Description:

A significantly higher proportion of patients with rare diseases (RD) with intellectual disability (ID), present hyperphagia, overweight or obesity, compared to the general population. Prader-Willi Syndrome (PWS) and related syndromes (PWS-like) represent the most well-known causes of eating disorders with early and severe obesity. Other known RD with ID have been described as being associated with eating disorders with overweight or obesity, which appear later in adolescence : Angelman's syndrome (approximately 40% of patients are overweight or obese, and 32% of children have hyperphagia), Fragile X syndrome (over 30% are obese), Smith-Magenis syndrome (50 to 60% are obese). Prader-Willi syndrome is the only genetic obesity identified to date associated with hyperghrelinemia, while ghrelin levels are lower than in controls in other situations of obesity.

The aim of the study is to find out whether the levels of ghrelin, which are abnormally high in PWS throughout life, are also high in these pathologies when people have hyperphagia and/or overweight.

The study involves a single visit carried out during a routine follow-up in the CRMR, in which the blood sample will allow the dosage of the ghrelin hormon. The visit will also involves a data collection and some questionnaires.

Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 300 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 1 Day
Official Title: Circulating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and / or Overweight, and / or Obesity
Estimated Study Start Date : March 15, 2021
Estimated Primary Completion Date : March 15, 2021
Estimated Study Completion Date : March 15, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rare Diseases

Groups and Cohorts
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Intervention Details:
  • Biological: acylated and unacylated ghrelin dosages
    realization of plasma samples to evaluate of levels of ghrelin and collection of plasma and cells

Outcome Measures
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Primary Outcome Measures :
  1. Levels of ghrelin in blood sample [ Time Frame: Day 1 ]
    dosage of ghrelin (pmol /l)


Secondary Outcome Measures :
  1. Overeating [ Time Frame: Day 1 ]
    Dykens overeating questionnaire

  2. Overeating [ Time Frame: Day 1 ]
    eating behavior assessment scale

  3. Behavioral disorder description [ Time Frame: Day 1 ]
    CBCL questionnaire for patients under 18 years old

  4. Behavioral disorder description [ Time Frame: Day 1 ]
    Developmental Behavior Checklist-Adult questionnaire for patients over 18 years old

  5. Social vulnerability of parents and / or legal guardians [ Time Frame: Day 1 ]
    EPICES questionnaire (Assessment of Precariousness and Health Inequalities for the Health Examination Centers).

  6. Family quality of life (for patients under 18) [ Time Frame: Day1 ]
    Parental-Developmental Disabilities Quality of Life questionnaire

  7. Burden of parents and / or legal guardians [ Time Frame: Day 1 ]
    ZBI questionnaire (Zarit Burden Interview).


Biospecimen Retention:   Samples Without DNA
collection de plasma et cellules sanguines

Eligibility Criteria
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Ages Eligible for Study:   3 Years to 50 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
patients infants and adults presenting a rare disease with overweight and hyperphagic behavior
Criteria

Inclusion Criteria:

  • Patients with one of the following rare diseases associated with : Angelman syndrome, Smith-Magenis syndrome, X Fragile syndrome, rare diseases of the cerebellum, rare epilepsies, PW-like syndromes or other rare diseases with eating disorders
  • Patients aged minimum 3 years and maximum 50 years.
  • Patients with overweight (or obesity) and/or hyperphagic behavior.

Exclusion Criteria:

  • Administrative problems: impossibility of giving parents or legal guardians informed information ; no coverage by a Social Security scheme.
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): NCT04768803


Contacts
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Contact: Nadege ALGANS 0561777204 algans.n@chu-toulouse.fr

Sponsors and Collaborators
University Hospital, Toulouse
Investigators
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Principal Investigator: Maithé TAUBER, MD University Hospital, Toulouse
More Information
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Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT04768803    
Other Study ID Numbers: RC31/19/0176
First Posted: February 24, 2021    Key Record Dates
Last Update Posted: February 24, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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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Intellectual Disability
Prader-Willi Syndrome
Angelman Syndrome
Smith-Magenis Syndrome
Syndrome
Overweight
Rare Diseases
Hyperphagia
Disease
Pathologic Processes
Obesity
Overnutrition
Nutrition Disorders
Body Weight
 Central Nervous System Diseases
Nervous System Diseases
Disease Attributes
Neurobehavioral Manifestations
Neurologic Manifestations
Neurodevelopmental Disorders
Mental Disorders
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Movement Disorders
Signs and Symptoms, Digestive
Chronobiology Disorders