Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome

What Was Tested

The trial (phase 2) is built around Oxytocin.

This study will utilize a randomized, placebo-controlled design for 12 weeks (phase 1), followed by an open-label extension for 12 weeks (phase 2).

The purpose is to evaluate the effect of intranasal oxytocin on impairments in attention, social memory, socialization, language, and repetitive behaviors.

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

The registry classes the interventions as drug.

Who Took Part

The registry lists 18 actual participants took part.

It was open to people of any sex aged from 5 years up to 17 years.

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

Examples of who could join: Have a diagnosis of Phelan-McDermid syndrome defined by pathogenic deletion or mutation of the SHANK3 gene and confirmed by chromosomal microarray or sequencing. Must be between the ages of 5 and 17. Must be on stable medication and psychosocial therapy regimens for at least three months prior to enrollment.

The full record lists 3 inclusion and 5 exclusion criteria; among the exclusions: allergy to oxytocin; active cardiovascular disease, epilepsy, or renal disease that is not controlled by medication; pregnant, lactating or refuse to practice contraception if sexually active.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

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

The Oxytocin group and the comparison group ran side by side rather than switching.

As a double-blind Oxytocin study, the assignments were hidden from both participants and investigators until the end.

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

It started in 2016-04-29 with completion listed for 2020-07-29.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Change in Aberrant Behavior Checklist (time frame: Baseline and Week 12).

It also tracked 12 secondary outcomes, for example Change in Visual Paired Comparison (VPC) Task, Change in Gap-Overlap Task, Change in Flicker Tasks, Change in Vineland Adaptive Behavior Scales, Change in Repetitive Behavior Scale-Revised (RBS-R) and Change in Clinical Global Impression - Severity Scales.

What This Means in Plain Language

Put simply, this is a phase 2, 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.