Oxytocin Trial in Prader-Willi Syndrome

What Was Tested

In this phase 1 trial, researchers set out to test Oxytocin.

The research questions are: 1.

Does intranasal oxytocin cause any side effects in children with PWS? 2.

It had two arms (groups): Intranasal oxytocin (active comparator) and Placebo (placebo comparator).

The registry classes the interventions as drug and other.

More on the Plan

This will be a randomized placebo controlled pilot study.

The primary outcome measure is to determine if intranasal administration of oxytocin will cause any adverse events in subjects with Prader-Willi syndrome.

Secondarily, the investigators will also perform evaluations to determine if intranasal oxytocin has any effect on social skills, behaviors, or appetite in children with Prader-Willi syndrome.

Who Took Part

Enrollment: 24 actual participants took part.

It was open to people of any sex aged from 5 years up to 11 years.

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

Examples of who could join: Children with genetically confirmed PWS. Ages at ≥ 5 years and ≤ 11 years (must start treatment prior to 11th birthday). Child must be in nutritional phase 2b or 3, as determined by the PI at each site. Must currently be on growth hormone treatment, and have been receiving growth hormone treatment for at least one year prior to screening date. Treatment cannot have been interrupted for more than 1 week within 3 months prior to screening date.

The full record lists 6 inclusion and 10 exclusion criteria; among the exclusions: inability to tolerate intranasal administration of medication; hepatic insufficiency (ast/alt greater than 3 times the normal levels for age); renal insufficiency (bun/creatinine greater than 3 times the normal levels for age).

How the Study Was Run

Design: randomized, crossover and quadruple-blind.

The participants were allocated by chance, so the Oxytocin group and the comparison group should start out alike.

In the crossover set-up each of the participants took both Oxytocin and the comparison in turn, acting as their own control.

Blinding on both sides kept the participants and researchers unaware of who received Oxytocin.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2015-03 with completion listed for 2015-08.

It ran at 3 sites in United States (3).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Safety of intranasal oxytocin in children with Prader-Willi syndrome (time frame: 3 months).

It also tracked 1 secondary outcome, for example Evaluation of food intake in Prader-Willi syndrome.

What This Means in Plain Language

Put simply, this is a phase 1, randomized trial of Oxytocin in 24 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.