Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
Overview
- Phase
- Phase 2
- Intervention
- Oxytocin
- Conditions
- Phelan-McDermid Syndrome
- Sponsor
- Alexander Kolevzon
- Enrollment
- 18
- Locations
- 1
- Primary Endpoint
- Change in Aberrant Behavior Checklist
- Status
- Completed
- Last Updated
- 4 years ago
Overview
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.
Investigators
Alexander Kolevzon
Alexander Kolevzon, Clinical Director
Icahn School of Medicine at Mount Sinai
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Oxytocin
The first phase of the study will follow a double-blind, placebo-controlled design. Participants randomized to the experimental group will receive intranasal oxytocin in doses of 24 IU, two times daily, for a total of 48 IU. Doses may be reduced by 8 IU/day if safety concerns emerge. During the second phase of the study, all participants will receive oxytocin, in identical doses.
Intervention: Oxytocin
Saline
During the first phase, patients randomized to the placebo group will receive intranasal saline solution in doses of 24 IU two times daily, for a total of 48 IU. During the second phase of the study, all participants will receive oxytocin, in identical doses.
Intervention: Saline
Outcomes
Primary Outcomes
Change in Aberrant Behavior Checklist
Time Frame: Baseline and Week 12
Change in scale at week 12 as compared to baseline. Aberrant Behavior Checklist - behavior rating subscales for the assessment of treatment effects.16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total score range from 0 to 48, with higher score indicating poorer health outcomes.
Secondary Outcomes
- Change in Flicker Tasks(Baseline and Week 12)
- Change in Vineland Adaptive Behavior Scales(Baseline and Week 12)
- Change in Repetitive Behavior Scale-Revised (RBS-R)(Baseline and Week 12)
- Change in Visual Paired Comparison (VPC) Task(Baseline and Week 12)
- Change in Gap-Overlap Task(Baseline and Week 12)
- Change in Clinical Global Impression - Severity Scales(Baseline and Week 12)
- Change in Short Sensory Profile (SSP)(Baseline and Week 12)
- Change in Macarthur-Bates Communicative Development Inventory (MCDI)(Baseline and Week 12)
- Change in EEG Tasks: Auditory Habituation(Baseline and Week 12)
- Number of Adverse Events(week 12)
- Change in Caregiver Strain Index(Baseline and Week 12)
- Change in Mullen Scales of Early Learning (MSEL)(baseline and 12 weeks.)