A Prospective, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Effectiveness of Intranasal Carbetocin in Subjects With Prader-Willi Syndrome (PWS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 38
- 试验地点
- 3
- 主要终点
- Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15)
研究概览
简要总结
The purpose of this study is to evaluate the safety and effectiveness of intranasal FE 992097 in children and adults with Prader-Willi Syndrome.
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
- Double
入排标准
- 年龄范围
- 10 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female 10-18 years of age (both inclusive)
- •Genetically confirmed diagnosis of Prader-Willi Syndrome
- •Determined to be in nutritional phase 3 Prader-Willi Syndrome based on Miller et al, 2011
排除标准
- •Known genetic, hormonal, or chromosomal cause of cognitive impairment other than Prader-Willi Syndrome
- •Presence of currently active psychotic symptoms
- •Presence of any cardiovascular disorders, epilepsy, frequent migraines or severe asthma
- •Previous diagnosis of autism spectrum disorder by a qualified healthcare provider
- •Prior or concomitant use of a selective serotonin reuptake inhibitor (SSRI) or selective norepinephrine reuptake inhibitor (SNRI), antipsychotic medication, wakefulness-promoting drug, or thyroid hormone unless dosage has been stable ≥6 months at time of screening
研究组 & 干预措施
Carbetocin (FE 992097)
干预措施: FE 992097 (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change From Baseline in Hyperphagia in Prader-Willi Syndrome (PWS) Questionnaires- Responsiveness (HPWSQ-R) Total Score at End-of-treatment (Day 15)
时间窗: From Day 1 (baseline) to Day 15
The HPWSQ-R is an 11-item questionnaire examining the psychological, developmental, and neurobiological correlates of hyperphagia in PWS. The items are classified into 3 domains; behavior, drive, and severity with each item rated on a five-point scale (1: not at all/none of the time/extremely easy to 5: extremely/all of the time/extremely hard). The questionnaire was completed by the parent/caregiver using a 1-week recall period. Total score was the sum of all the items in the three domains and ranged from 11 (no hyperphagia behaviors) to 55 (most severe hyperphagia behaviors). Change from baseline in HPWSQ-R Total Score at Day 15 is presented for this outcome measure.
次要结局
- Clinical Global Impression- Improvement After Treatment (CGI-I) Score at End-of-treatment (Day 15)(At Day 15)
- Change From Baseline in HPWSQ-R Domain Scores (Behavior, Drive and Severity) at End-of-treatment (Day 15)(From Day 1 (baseline) to Day 15)
- Change From Baseline in Children's Yale-Brown Obsessive Compulsive Scale Score (CY-BOCS) at End-of-treatment (Day 15)(From Day 1 (baseline) to Day 15)
- Change From Baseline in the Food Domain Score of the Reiss Profile at End-of-treatment (Day 15)(From Day 1 (baseline) to Day 15)