PWS European Blood Bank for Infants and Controls From 0 to 48 Months
- Conditions
- Prader-Willi Syndrome
- Interventions
- Other: blood samples
- Registration Number
- NCT02529085
- Lead Sponsor
- University Hospital, Toulouse
- Brief 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.
- Detailed 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 & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 215
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description control group blood samples Blood samples for the bank in children hospitalized for a planned surgery for malformation, orthopaedic or visceral surgery Infants with PWS blood samples Blood samples for the bank in link with a multicenter database including clinical data on birth, auxology, endocrine functions and feeding behaviour
- Primary Outcome Measures
Name Time Method Levels of hormones and neuropeptides 42 months Measure of hormones and neuropeptides (ghrelin, insulin, leptin, pancreatic polypeptide, oxytocin, melatonin, orexins) involved in feeding and appetite regulation
- Secondary Outcome Measures
Name Time Method Correlation between hormones and neuropeptides levels 42 months study the correlation between ghrelin, insulin, leptin, pancreatic polypeptide, oxytocin, melatonin, orexins
Trial Locations
- Locations (6)
Karolinska University Hospital
🇸🇪Stockholm, Sweden
Department of Pediatrics / Division of Endocrinology / Erasmus University Medical Center / Sophia Children's Hospital Rotterdam
🇳🇱Rotterdam, Netherlands
Unité d'Endocrinologie Pédiatrique / Université Catholique de Louvain
🇧🇪Brussels, Belgium
Department of Endocrinology / University Children's Hospital
🇩🇪Essen, Germany
Department of Pediatrics / Division of Endocrinology
🇫🇷Toulouse, Haute-Garonne, France
Metabolic & Molecular Imaging Group / MRC Clinical Sciences Centre / Imperial College London / Hammersmith Hospital
🇬🇧London, United Kingdom