Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants

What Was Tested

The trial (phase 1, phase 2) is built around Oxytocin.

The Prader-Willi syndrome (PWS) 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 hyperphagia and deficit of satiety, 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.

Swaab reported a deficit in the oxytocin (OT)-producing neurons of the paraventricular nucleus in the brain of these patients.

It had three arms (groups): first arm (active comparator), second arm (active comparator) and third arm (active comparator).

The registry classes the interventions as drug.

More on the Plan

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 hyperphagia and deficit of satiety, combined with other endocrine dysfunction probably due to hypothalamic dysfunction.

Who Took Part

Enrollment: 18 actual participants took part.

It was open to people of any sex aged from 1 Month up to 5 months.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Infants with PWS genetically confirmed. Aged less than 5 months.

The full record lists 2 inclusion and 3 exclusion criteria; among the exclusions: infants presenting hepatic insufficiency; infants presenting renal insufficiency; infants with abnormal ecg.

How the Study Was Run

Design: non-randomized, parallel-group and open-label.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Nothing was hidden in this open-label Oxytocin study: the participants and staff both knew the treatment.

It started in 2013-05 with completion listed for 2014-07.

It ran at 1 site in France.

Its stated primary purpose is treatment.

What Was Measured

It also tracked 2 secondary outcomes, for example NOMAS Score and Videofluoroscopy of Swallowing Score.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, non-randomized trial of Oxytocin in 18 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Oxytocin compound page, all human trials.