Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome

Summary

This is a pilot study examining the efficacy, safety and tolerability of intranasal oxytocin as a novel treatment in Phelan-McDermid syndrome (PMS). 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.

Timeline

Start

2016-04-29

Primary completion

2020-07-29

Completion

2020-07-29

Outcome

Missed primary endpoint

paper There was no statistically significant improvement with oxytocin as compared to placebo on the ABC-SW (Mann-Whitney U = 50, p = 0.055) PMID 34593045 ↗

Publications

• Results Fastman J, Foss-Feig J, Frank Y, Halpern D, Harony-Nicolas H, Layton C, Sandin S, Siper P, Tang L, Trelles P, Zweifach J, Buxbaum JD, Kolevzon A. A randomized controlled trial of intranasal oxytocin in Phelan-McDermid syndrome. Mol Autism. 2021 Sep 30;12(1):62. doi: 10.1186/s13229-021-00459-1.

Drugs

Indications