Clinical Trial: Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome

Brief 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.

Detailed Summary:
Sponsor: Alexander Kolevzon

Current Primary Outcome: Aberrant Behavior Checklist - Social Withdrawal [ Time Frame: Baseline and Week 12 ]

Change in scale at week 12 as compared to baseline. Aberrant Behavior Checklist - Social Withdrawal Subscale - a behavior rating scale for the assessment of treatment effects.


Original Primary Outcome: Computerized eye tracking tasks to measure attention [ Time Frame: Baseline and Week 12 ]

Change in scale at week 12 as compared to baseline. Visual Paired Comparison, Gap-Overlap, and Flicker tasks


Current Secondary Outcome:

  • Visual Paired Comparison (VPC) Task [ Time Frame: Baseline and Week 12 ]
    Change in scale at week 12 as compared to baseline. The subject is initially presented with a target for familiarization and the familiar target is then paired with a novel target. Recognition is indexed by a novelty score derived from the percentage of time looking at the novel stimulus vs. the familiar stimulus.
  • Gap-Overlap Task [ Time Frame: Baseline and Week 12 ]
    One of three computerized eye tracking tasks to measure attention. three computer screens are set up side by side. A stimulus initially appears on the central screen, and once the participant orients to that stimulus, another stimulus will appear on one of the lateral screens. In the "gap" condition, the central stimulus will disappear before the peripheral stimulus appears. In the "overlap" condition, the central stimulus will remain on the screen while the peripheral stimulus appears. In both conditions, reaction times of the participant's eye movements (saccadic reaction time) from the central stimulus to the peripheral stimulus will be measured.
  • Flicker tasks [ Time Frame: Baseline and Week 12 ]
    One of three computerized eye tracking tasks to measure attention. The "flicker task," or the four-alternative forced-choice preferential looking paradigm - the time interval over which the visual system is able to parse information.
  • Vineland Adaptive Behavior Scales [ Time Frame: Baseline and Week 12 ]
    Change in scale at week 12 as compared to baseline. Vineland Adaptive Behavior Communication Domain measures adaptive functioning.
  • Repetitive Behavior Scale-Revised (RBS-R) [ Time Frame: Baseline and Week 12 ]
    Change in Repetitive Behavior Scale at week 12 as compared to baseline.
  • Clinical Global Impression-Improvement and Severity Scales [ Time Frame: Baseline and Week 12 ]
    Clinical Global Impression-Improvement and Severity Scales at week 12 as compared to baseline.
  • Sensory Profile [ Time Frame: Baseline and Week 12 ]
    Sensory Profile Scale at week 12 as compared to baseline, measuring sensory sensitivity and sensory seeking behavior.
  • Macarthur-Bates Communicative Development Inventory [ Time Frame: Baseline and Week 12 ]
    Macarthur-Bates Communicative Development Inventory at week 12 as compared to baseline, measuring language.
  • Electrophysiology Recording [ Time Frame: Baseline and Week 12 ]
    Change at 12 weeks as compared to baseline using electroencephalographic recordings to measure Visual Evoked and Auditory Event Related Potentials


Original Secondary Outcome:

  • Aberrant Behavior Checklist - Social Withdrawal [ Time Frame: Baseline and Week 12 ]
    Change in scale at week 12 as compared to baseline. Aberrant Behavior Checklist - Social Withdrawal Subscale - a behavior rating scale for the assessment of treatment effects.
  • Vineland Adaptive Behavior Scales [ Time Frame: Baseline and Week 12 ]
    Change in scale at week 12 as compared to baseline. Vineland Adaptive Behavior Communication Domain measures adaptive functioning.
  • Repetitive Behavior Scale-Revised (RBS-R) [ Time Frame: Baseline and Week 12 ]
    Change in Repetitive Behavior Scale at week 12 as compared to baseline.
  • Clinical Global Impression-Improvement and Severity Scales [ Time Frame: Baseline and Week 12 ]
    Clinical Global Impression-Improvement and Severity Scales at week 12 as compared to baseline.
  • Sensory Profile [ Time Frame: Baseline and Week 12 ]
    Sensory Profile Scale at week 12 as compared to baseline, measuring sensory sensitivity and sensory seeking behavior.
  • Macarthur-Bates Communicative Development Inventory [ Time Frame: Baseline and Week 12 ]
    Macarthur-Bates Communicative Development Inventory at week 12 as compared to baseline, measuring language.
  • Electrophysiology Recording [ Time Frame: Baseline and Week 12 ]
    Change at 12 weeks as compared to baseline using electroencephalographic recordings to measure Visual Evoked and Auditory Event Related Potentials


Information By: Icahn School of Medicine at Mount Sinai

Dates:
Date Received: March 7, 2016
Date Started: April 2016
Date Completion: June 2018
Last Updated: November 2, 2016
Last Verified: November 2016