Oxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years

What Was Tested

In this not phased trial, researchers set out to test Oxytocin.

Positive results in preclinical and clinical studies in adults and infants with Prader-Willi syndrome lead investigators to set up a new study in children with Prader-Willi syndrome.

The objective of this study is to document effects of oxytocin intranasal administrations on behavioural troubles in children with Prader-Willi syndrome aged from 3 to 12 years.

It had two arms (groups): Oxytocin (experimental) and Placebo (placebo comparator).

The registry classes the interventions as drug.

More on the Plan

Two groups of patients will be constituted according to their age; "Group 1" children aged from 3 to 6 years (n = 20) and "Group 2" children aged from 7 to 12 years (n = 20).

Within each group, subjects will be randomized to receive either oxytocin or placebo for 12 consecutive weeks.

A second period of 12 consecutive oxytocin treatment weeks will then be performed for all patients, followed by a 4-week observation period to document effects after discontinuation of treatment.

Who Took Part

Enrollment: 40 actual participants took part.

It was open to people of any sex aged from 3 years up to 12 years.

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

Examples of who could join: Patient with a complete genetic diagnosis of Prader-Willi syndrome. Patient treated by growth hormone for at least 1 year. Patient naïve for oxytocin for at least 5 years.

The full record lists 3 inclusion and 6 exclusion criteria; among the exclusions: patient who do not accept intranasal administrations (major behavioural trouble); patient with hepatic insufficiency : serum transaminases (sgot, sgpt) higher than 3 times normal values for age; patient with renal insufficiency : serum creatinine higher than 3 times normal values for age.

How the Study Was Run

The set-up was randomized, parallel-group and double-blind.

Because people in this Oxytocin study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

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

Neither the participants nor the staff measuring them knew who was on Oxytocin, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant and investigator.

It started in 2016-11-28 with completion listed for 2019-01.

It ran at 1 site in France.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Evolution of behavioural troubles evaluated by the global score of Child Behavior Check List Questionnaire after 12 weeks of oxytocin/placebo treatment (time frame: Week 12).

It also tracked 8 secondary outcomes, for example Evaluation of hyperphagia after 12 weeks of oxytocin/placebo treatment, Evaluation of social skills after 12 weeks of oxytocin/placebo treatment, Evaluation of auto- and hetero-aggressive after 12 weeks of oxytocin/placebo treatment, Evaluation of psychopathology after 12 weeks of oxytocin/placebo treatment, Evaluation of global clinical status after 12 weeks of oxytocin/placebo treatment and Evaluation of acyl and desacyl ghrelin plasma levels after 12 weeks of oxytocin/placebo treatment.

What This Means in Plain Language

Put simply, this is a not phased, randomized trial of Oxytocin in 40 people.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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