PWS European Blood Bank for Infants and Controls From 0 to 48 Months

Prader-Willi Syndrome Clinical Trial

Official title:

Study of Early Endocrine Profile in Infants With Prader-Willi Syndrome (PWS) in Order to Unravel the Switch From Early Feeding Difficulties to Obesity and Hyperphagia.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02529085
• Other study ID #: 12 359 03
• Status: Completed
• Phase: N/A
• Start date: March 2013
• Est. completion date: June 1, 2017

Study information

• Verified date: November 2020
• Source: University Hospital, Toulouse
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The present project aims to determine the underlying mechanisms for the switch from failure to thrive to excessive weight gain and hyperphagia with impaired satiety in PWS. The primary objective is to describe the evolution of circulating hormones involved in feeding and appetite regulation during the 4 first years of life. The secondary objective is to make this blood bank available for other research projects and particularly the investigation of hormones involved in hypogonadism.

Over the last ten years, the age at diagnosis in PWS has fallen significantly and the majority of cases is now diagnosed during the 1st trimester of life giving the possibility to collect precise clinical data and serum samples at early stages. The investigators of the project are involved in the care of patients with PWS and have a devoted clinic and an organized network in their country through clinical networks or patient associations.

Description:

The investigators propose to perform a prospective multicentric study, both longitudinal (duration 30 months) and cross-sectional with implementation of a blood bank in link with a multicenter database including clinical data on birth, auxology, endocrine functions and feeding behaviour. The cohort will include 200 infants from 3 to 48 months with PWS and 200 controls matched on age recruited in the 6 participating countries. The investigators make the assumption that 3 blood sampling will be necessary during the first year and 6 monthly sampling thereafter. For measuring hormones and neuropeptides (ghrelin, insulin, leptin, pancreatic polypeptide, oxytocin, cortisol, melatonin, orexin A, GLP-1 and PYY) involved in feeding and appetite regulation the investigators will use primarily multiplex microplates technics requiring 50-200µl of sample. Intragroup and intergroup comparisons will be performed in order to describe the evolution of each hormone with time and to compare data obtained in the PWS group with those obtained in the control group.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 215
• Est. completion date: June 1, 2017
• Est. primary completion date: June 1, 2017
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria (PWS infants cohort)

• Genetic diagnosis of Prader-Willi syndrome

Exclusion Criteria (PWS infants cohort)

• none

Inclusion Criteria (control group)

• children hospitalized for a planned surgery for malformation, orthopaedic or visceral surgery

Exclusion Criteria (control group)

• children with endocrine disorder

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Other:
• blood samples
blood samples for the bank

Locations

Country	Name	City	State
Belgium	Unité d'Endocrinologie Pédiatrique / Université Catholique de Louvain	Brussels
France	Department of Pediatrics / Division of Endocrinology	Toulouse	Haute-Garonne
Germany	Department of Endocrinology / University Children's Hospital	Essen
Netherlands	Department of Pediatrics / Division of Endocrinology / Erasmus University Medical Center / Sophia Children's Hospital Rotterdam	Rotterdam
Sweden	Karolinska University Hospital	Stockholm
United Kingdom	Metabolic & Molecular Imaging Group / MRC Clinical Sciences Centre / Imperial College London / Hammersmith Hospital	London

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital, Toulouse	European Society for Paediatric Endocrinology

Countries where clinical trial is conducted

Belgium,  France,  Germany,  Netherlands,  Sweden,  United Kingdom, 

References & Publications (1)

Cadoudal T, Buléon M, Sengenès C, Diene G, Desneulin F, Molinas C, Eddiry S, Conte-Auriol F, Daviaud D, Martin PG, Bouloumié A, Salles JP, Tauber M, Valet P. Impairment of adipose tissue in Prader-Willi syndrome rescued by growth hormone treatment. Int J Obes (Lond). 2014 Sep;38(9):1234-40. doi: 10.1038/ijo.2014.3. Epub 2014 Jan 10. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Levels of hormones and neuropeptides	Measure of hormones and neuropeptides (ghrelin, insulin, leptin, pancreatic polypeptide, oxytocin, melatonin, orexins) involved in feeding and appetite regulation	42 months
Secondary	Correlation between hormones and neuropeptides levels	study the correlation between ghrelin, insulin, leptin, pancreatic polypeptide, oxytocin, melatonin, orexins	42 months