Piloting Treatment With Intranasal Oxytocin in Phelan-McDermid Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Change in Aberrant Behavior Checklist
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
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.
干预措施: Oxytocin (Drug)
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.
干预措施: Saline (Drug)
结局指标
主要结局
Change in Aberrant Behavior Checklist
时间窗: 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.
次要结局
- 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.)
研究者
Alexander Kolevzon
Alexander Kolevzon, Clinical Director
Icahn School of Medicine at Mount Sinai