Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome

What Was Tested

In this phase 2 trial, researchers set out to test Oxytocin for hyperparathyroidism.

The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels.

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

The registry classes the interventions as drug and other.

More on the Plan

The investigators propose a randomized double-blind 8 week treatment trial of intranasal OXT vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2) Repetitive and disruptive behaviors and (3) Salivary OXT levels.

Who Took Part

23 actual participants took part.

It was open to people of any sex aged from 5 years up to 18 years.

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

Examples of who could join: Male or Female child outpatients aged 5 to 18 years. Have a physical exam and laboratory results that are within the norms for PWS.

The full record lists 5 inclusion and 7 exclusion criteria; among the exclusions: exposure to any investigational agent in the 30 days prior to randomization; prior chronic treatment with oxytocin.

How the Study Was Run

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

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

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

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

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

It started in 2015-10 with completion listed for 2016-12.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Hyperphagia Questionnaire (HQ)- Total Score (time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported). Hyperphagia Questionnaire (HQ)- Behavior Factor Score (time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported). Hyperphagia Questionnaire (HQ)- Drive Factor Score (time frame: Assessed trends over multiple time points (weeks 0, 2, 4, 6, and 8), week 0 (baseline), week 4 and week 8 are reported).

It also tracked 6 secondary outcomes, for example Repetitive Behavior Scale-Revised (RBS-R) Ritualistic Behavior Factor Score, Repetitive Behavior Scale-Revised (RBS-R) Sameness Factor Score, Repetitive Behavior Scale-Revised (RBS-R) Stereotyped Behavior Factor Score, Repetitive Behavior Scale-Revised (RBS-R) Compulsive Behavior Factor Score, Repetitive Behavior Scale-Revised (RBS-R) Self-Injurious Factor Score and Repetitive Behavior Scale-Revised (RBS-R) Restricted Interest Factor Score.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Oxytocin in 23 people with hyperparathyroidism.

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, Hyperparathyroidism research.