Phase 3, Randomized, Double-Blind, Placebo-Controlled, 8-week Clinical Study to Assess the Efficacy, Safety, and Tolerability, of Intranasal Carbetocin (LV-101) in Prader-Willi Syndrome (PWS) With Long Term Follow-Up (CARE-PWS)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 130
- 试验地点
- 24
- 主要终点
- Hyperphagia Behavior
研究概览
简要总结
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin). This study will also evaluate the safety and tolerability of LV-101.
详细描述
This is a Phase 3 randomized, double-blind study with an 8-week, placebo-controlled period designed to test the effectiveness, safety, and tolerability of LV-101 in participants with PWS.
Effectiveness will be measured using both caregiver-reported and clinician-reported measures of hyperphagia (extreme hunger), obsessive and compulsive behaviors, and anxiety. Safety and tolerability will be measured by adverse events, laboratory tests, and physical exams.
After the 8-week placebo-controlled period, there will be a long-term follow-up period of 56 weeks and an optional extension period after study week 64 during which all participants will receive active treatment with LV-101. At Week 8, participants who were randomized to placebo in the placebo-controlled period will be randomized to one of the two LV-101 doses, administered three times per day before meals.
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
- 7 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically-confirmed Prader-Willi syndrome
- •Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
- •PWS Nutritional Phase 3 (hyperphagic, rarely feels full)
排除标准
- •Living in a group home
- •Genetically diagnosed Schaaf-Yang syndrome or other genetic, hormonal, or chromosomal cognitive impairment
- •New food-related interventions, including environment or dietary restrictions, within 1 month of screening
- •Dose of any allowed chronic concomitant medications or supplements that have not been stable for ≥3 months prior to the study or is not expected to remain stable while participating in the study; adjustments in growth hormone dose ≤10% are not exclusionary
- •Presence of cardiovascular disorders, epilepsy, frequent migraines, or severe asthma
- •More than 3 episodes of sinusitis in the 12 months prior to Screening Visit or presence of nasal diseases that may affect deposition of intranasal medication
- •Unwilling to abstain from nasal saline, other nasal irrigation, or other intranasal medications for 2 weeks prior to the Baseline visit and during the 8-week, placebo-controlled period of the study
- •Use of weight loss medication, oxytocin, carbetocin, or vasopressin in the 6 months prior to screening
- •Participation in an interventional research study involving another investigational medication or device in the 6 months prior to screening or during the study
- •Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to unstable medical condition, inability to comply with the protocol, or other risk to subject or to the integrity of the study
研究组 & 干预措施
Placebo
matched placebo during first 8-weeks; prospectively randomized 1:1 to either one of the two doses of carbetocin during 56-week follow-up and optional extension periods
干预措施: placebo (Drug)
3.2 mg of LV-101
3.2 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods
干预措施: 3.2 mg intranasal carbetocin (Drug)
9.6 mg of LV-101
9.6 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods
干预措施: 9.6 mg intranasal carbetocin (Drug)
结局指标
主要结局
Hyperphagia Behavior
时间窗: Baseline to Week 8
Change in hyperphagia (extreme hunger) as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Total Score versus placebo. Score range: 0-36; higher scores mean a worse outcome. Reduction in score indicates improvement.
Obsessive and Compulsive Behaviors
时间窗: baseline to Week 8
Change in obsessive and compulsive behaviors as measured by the Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) Total Score versus placebo. Score range: 0-40; higher scores mean a worse outcome. Reduction in score indicates improvement.
次要结局
- Anxiety(Baseline to Week 8)
- Hyperphagia Behavior (Subset)(Baseline to Week 8)
- Global Impression(Week 8)