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Tolerance of Intranasal Administration of OT in Prader-Willi Newborn Babies

NCT01548521 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2017-02-23

No results posted yet for this study

Summary

Background: Prader-Willi syndrome (PWS) is a rare, complex multisystem genetic disorder arising from the lack of expression of paternally inherited imprinted genes on chromosome 15q11-q13. The syndrome includes severe neonatal hypotonia with impaired suckling leading to failure to thrive in the most severe cases, subsequently followed by an early onset of morbid obesity with insatiable hunger, combined with other endocrine dysfunction probably due to hypothalamic dysfunction. The pathophysiological mechanism of the occurrence of the 2 main nutritional phases of PWS is unknown. A deficit in the oxytocin (OT)-producing neurons of the paraventricular nucleus in the brain of these patients has been reported. In addition of its well-known anorexigenic effect, OT is involved in establishing and maintaining social codes. Indeed, we have recently shown in a double blind placebo study, that OT administration to adult patients with PWS significantly decreased depressive mood tendencies and tantrums while increasing trust in others with some data on a trend to decrease appetite with higher satiety. Moreover in a PWS mouse model generated from a MAGEL2 KO gene a single OT injection at 5 hr of life prevent the early death observed in 50 % of the new born mice by recovering normal suckling. Interestingly this effect is no longer observed if OT injection takes place later. These data, OT deficit in PWS, good tolerance of OT and its effect after intranasal administration in adult patients with PWS and the recent striking data obtained in the MAGEL2 mouse model, prompted us to evaluate the tolerance of a single administration of intranasal OT in PWS newborns and its possible effect on suckling and food intake. Nowadays the diagnosis of PWS is done during the first months of life in our country. At this age, children still present with poor suckling suggesting that OT may be still efficient. Moreover in adult patients with PWS we have shown that OT improves some typical behavioral troubles. Therefore we first want to evaluate the tolerance of the intranasal administration of OT in 6 infants with PWS genetically confirmed and its effect on suckling, milk intake and weight gain.

Conditions

Interventions

DRUG

Oxytocin

2 ui intranasal administration for the 3 first patients, 4UI for the 3 following patients.

Sponsors & Collaborators

  • University Hospital, Toulouse

    lead OTHER

Principal Investigators

  • Maithe TAUBER, MD · Hospital of Toulouse

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
15 Days
Max Age
5 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-07-31
Primary Completion
2012-02-29
Completion
2012-04-30

Countries

  • France

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01548521 on ClinicalTrials.gov