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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

Tracking Information
First Submitted Date February 16, 2021
First Posted Date February 24, 2021
Last Update Posted Date February 24, 2021
Estimated Study Start Date March 15, 2021
Estimated Primary Completion Date March 15, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: February 23, 2021)		 Levels of ghrelin in blood sample [ Time Frame: Day 1 ]
dosage of ghrelin (pmol /l)
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures
 (submitted: February 23, 2021)
  • Overeating [ Time Frame: Day 1 ]
    Dykens overeating questionnaire
  • Overeating [ Time Frame: Day 1 ]
    eating behavior assessment scale
  • Behavioral disorder description [ Time Frame: Day 1 ]
    CBCL questionnaire for patients under 18 years old
  • Behavioral disorder description [ Time Frame: Day 1 ]
    Developmental Behavior Checklist-Adult questionnaire for patients over 18 years old
  • 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).
  • Family quality of life (for patients under 18) [ Time Frame: Day1 ]
    Parental-Developmental Disabilities Quality of Life questionnaire
  • Burden of parents and / or legal guardians [ Time Frame: Day 1 ]
    ZBI questionnaire (Zarit Burden Interview).
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and/or Overweight, and/or Obesity
Official Title Circulating Levels of Ghrelin in Patients With a Rare Disease Associated With Intellectual Disability, and Hyperphagia, and / or Overweight, and / or Obesity
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.
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 Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 1 Day
Biospecimen Retention:   Samples Without DNA
Description:
collection de plasma et cellules sanguines
Sampling Method Non-Probability Sample
Study Population patients infants and adults presenting a rare disease with overweight and hyperphagic behavior
Condition
  • Angelman Syndrome
  • Smith-Magenis Syndrome
  • X Fragile Syndrome
  • Epilepsy
  • Prader-Willi Syndrome
Intervention Biological: acylated and unacylated ghrelin dosages
realization of plasma samples to evaluate of levels of ghrelin and collection of plasma and cells
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Unknown status
Estimated Enrollment
 (submitted: February 23, 2021)		 300
Original Estimated Enrollment Same as current
Estimated Study Completion Date March 15, 2022
Estimated Primary Completion Date March 15, 2021   (Final data collection date for primary outcome measure)
Eligibility 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.
Sex/Gender
Sexes Eligible for Study: All
Ages 3 Years to 50 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number NCT04768803
Other Study ID Numbers RC31/19/0176
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Current Responsible Party University Hospital, Toulouse
Original Responsible Party Same as current
Current Study Sponsor University Hospital, Toulouse
Original Study Sponsor Same as current
Collaborators Not Provided
Investigators
Principal Investigator: Maithé TAUBER, MD University Hospital, Toulouse
PRS Account University Hospital, Toulouse
Verification Date February 2021