Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.

What Was Tested

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

The objective of this study is to collect data on tolerance and effects of early treatment with oxytocin in children with Prader Willi Syndrome aged from 3 to 4 years and to compare these infants with not treated age-matched infants with Prader Willi Syndrome.

It had two arms (groups): Oxytocin (experimental) and Control (experimental).

Groups of children with Prader Willi Syndrome treated by oxytocin for 7 days during their first 6 months of life.

Groups of children with Prader Willi Syndrome not treated by oxytocin for 7 days during their first 6 months of life.

The registry classes the interventions as drug and other.

More on the Plan

In accordance with recommendations of regulatory authorities, the researchers want to collect long term data of patients treated with oxytocin before the age of 6 months.

Moreover clinical observations of these infants support long term effects on communication skills, global development and behaviour.

Who Took Part

34 actual participants took part.

It was open to people of any sex aged from 3 years up to 4 years.

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

Examples of who could join: Infants with Prader Willi Syndrome (genetic diagnosis confirmed). For treated group : infant included in the ancient study. For not treated group: infant never treated with oxytocin.

The full record lists 3 inclusion and 4 exclusion criteria; among the exclusions: subject involved in another search including an exclusion period still in progress at the time of inclusion; impossibility to give parents or legal guardian informed information; no coverage by a social security scheme.

How the Study Was Run

The set-up was non-randomized, parallel-group and open-label.

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

Everyone knew when Oxytocin was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

It started in 2017-01 with completion listed for 2018-05.

It ran at 1 site in France.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Evaluation of communication skills (time frame: Day 1).

It also tracked 6 secondary outcomes, for example Evaluation of adaptative behavior composite and 3 domains : "Daily living skills", "Socialization", "Motor skills", Evaluation of behavioral troubles, Evaluation of global development, Evaluation of orality and eating behaviour, Evaluation of brain activity and Evaluation of plasma levels of ghrelin and other peptides involved in feeding behaviour or energy metabolism.

What This Means in Plain Language

Put simply, this is a not phased, non-randomized trial of Oxytocin in 34 people.

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

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.