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

NCT ID: NCT02529085

Last Updated: 2020-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

215 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-03-31

Study Completion Date

2017-06-01

Brief Summary

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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

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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.

Conditions

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Prader-Willi Syndrome

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

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Infants with PWS

Blood samples for the bank in link with a multicenter database including clinical data on birth, auxology, endocrine functions and feeding behaviour

Group Type EXPERIMENTAL

blood samples

Intervention Type OTHER

blood samples for the bank

control group

Blood samples for the bank in children hospitalized for a planned surgery for malformation, orthopaedic or visceral surgery

Group Type OTHER

blood samples

Intervention Type OTHER

blood samples for the bank

Interventions

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blood samples

blood samples for the bank

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Genetic diagnosis of Prader-Willi syndrome


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

Exclusion Criteria

* none


* children with endocrine disorder
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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European Society for Paediatric Endocrinology

UNKNOWN

Sponsor Role collaborator

University Hospital, Toulouse

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Maithe TAUBER, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Toulouse

Locations

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Unité d'Endocrinologie Pédiatrique / Université Catholique de Louvain

Brussels, , Belgium

Site Status

Department of Pediatrics / Division of Endocrinology

Toulouse, Haute-Garonne, France

Site Status

Department of Endocrinology / University Children's Hospital

Essen, , Germany

Site Status

Department of Pediatrics / Division of Endocrinology / Erasmus University Medical Center / Sophia Children's Hospital Rotterdam

Rotterdam, , Netherlands

Site Status

Karolinska University Hospital

Stockholm, , Sweden

Site Status

Metabolic & Molecular Imaging Group / MRC Clinical Sciences Centre / Imperial College London / Hammersmith Hospital

London, , United Kingdom

Site Status

Countries

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Belgium France Germany Netherlands Sweden United Kingdom

References

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Cadoudal T, Buleon M, Sengenes C, Diene G, Desneulin F, Molinas C, Eddiry S, Conte-Auriol F, Daviaud D, Martin PG, Bouloumie 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.

Reference Type BACKGROUND
PMID: 24406482 (View on PubMed)

Beauloye V, Diene G, Kuppens R, Zech F, Winandy C, Molinas C, Faye S, Kieffer I, Beckers D, Nergardh R, Hauffa B, Derycke C, Delhanty P, Hokken-Koelega A, Tauber M. High unacylated ghrelin levels support the concept of anorexia in infants with prader-willi syndrome. Orphanet J Rare Dis. 2016 May 4;11(1):56. doi: 10.1186/s13023-016-0440-0.

Reference Type DERIVED
PMID: 27146407 (View on PubMed)

Other Identifiers

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12 359 03

Identifier Type: -

Identifier Source: org_study_id