Ecopipam 治疗 7-17 岁受试者图雷特综合症
研究概览
详细说明
研究类型
注册 (实际的)
阶段
- 阶段2
联系人和位置
学习地点
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California
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Los Angeles、California、美国、90024
- UCLA
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Florida
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St. Petersburg、Florida、美国、33701
- University of South Florida/Rothman Center for Neuropsychiatry
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Georgia
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Atlanta、Georgia、美国、30322
- Emory University School of Medicine
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Illinois
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Chicago、Illinois、美国、60637
- University of Chicago Medical School
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Kansas
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Kansas City、Kansas、美国、64108
- Children's Mercy Kansas City
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Missouri
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St Louis、Missouri、美国、63110
- Washington University School of Medicine
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New Jersey
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Summit、New Jersey、美国、07902
- Overlook Hospital
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New York
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Manhasset、New York、美国、11030
- North Shore-Long Island Jewish Hosptial
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New York、New York、美国、10065
- Weill Cornell Medical School
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Ohio
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Cincinnati、Ohio、美国、45228
- Cincinnati Children's Hospital
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Texas
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Houston、Texas、美国、77030
- Baylor College of Medicine
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
描述
入选标准： • 根据临床医生管理的 TS 诊断置信指数 (DCI)，受试者必须患有妥瑞氏综合症 (TS)。
- 受试者必须表现出运动和发声抽动。
- 受试者在耶鲁全球抽动严重程度量表上的筛选和基线（就在第一次治疗之前）的最低分数必须为 20。
- 受试者必须符合年龄（≥ 7 至 < 18 岁）
- 受试者体重必须≥ 20 公斤（45 磅）
- 性活跃的育龄女性必须使用有效的避孕措施（即口服避孕药、宫内节育器、避孕套和杀精子剂的双重屏障方法），并同意在参与研究期间继续使用避孕措施。 他们还必须同意在最后一次服用研究药物后 30 天内采取避孕措施。
- 性活跃的男性受试者必须在研究期间使用屏障避孕方法，并同意在最后一次研究药物给药后至少 30 天内继续使用男性避孕方法。
- 受试者的父母或法定监护人必须签署书面知情同意书。
- 受试者必须签署书面知情同意书。
排除标准：
- 在筛选时患有不稳定的内科疾病或在实验室测试或心电图上有临床显着异常的受试者。
- 受试者在过去 2 年内有过严重的抑郁发作
- 有自杀未遂史的受试者
- 具有临床显着自杀倾向的受试者（基于哥伦比亚自杀评定量表（C-SSRS）
- 一级亲属患有严重抑郁症并导致任何精神病住院或企图/完成自杀的受试者，但因产后抑郁症住院除外。
- 有癫痫发作史的受试者（不包括过去 2 年以上发生的热性惊厥）
- 6个月内发生心肌梗塞的受试者。
- 目前怀孕或哺乳期的女孩。
- 需要可能对 TS 症状产生影响的药物（ecopipam 除外）的受试者（即锂、精神兴奋剂）
- 需要与 ecopipam 有不利相互作用的药物的受试者，例如多巴胺拮抗剂或激动剂[包括安非他酮]、丁苯那嗪或单胺氧化酶抑制剂。
- 具有 I 型或 II 型双相情感障碍、痴呆、精神分裂症或精神障碍诊断和统计手册结构化临床访谈 - 第 4 版 (DSM-IV) Axis-I 障碍 (SCID) 确定的终生病史的受试者.
- 当前或近期（过去 3 个月）滥用或依赖 DSM-IV 物质（尼古丁除外）的受试者。
- 筛选时尿液药物筛查（可卡因、苯丙胺、甲基苯丙胺、四氢大麻酚 (THC)、苯二氮卓类药物、巴比妥类药物、苯环利定 (PCP)、阿片类药物）呈阳性的受试者。 尿液仅对苯二氮卓类药物和/或大麻呈阳性的受试者（即，根据 DSM-IV 标准，使用者而非滥用者）可能符合条件。
- 先前接受过 ecopipam 治疗的受试者。
接受过以下治疗的受试者：
- 研究开始后 3 个月内的研究药物
- 在开始研究后 3 个月内储存抗精神病药
- 筛选前 2 周内可能对 TS 症状产生影响的其他精神药物（即锂、丁苯那嗪）。
- 4周内口服抗精神病药
- 选择性 5-羟色胺再摄取抑制剂，除非在研究开始前剂量稳定至少 4 周，并且未规定用于缓解 TS 的神经体征
学习计划
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：交叉作业
- 屏蔽：四人间
武器和干预
参与者组/臂 |
干预/治疗 |
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实验性的：Ecopipam/Placebo
Given Ecopipam first and then crossed over to Placebo
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Ecopipam 是多巴胺 D1 受体家族的选择性拮抗剂。
其他名称：
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实验性的：Placebo/Ecopipam
Given placebo first and crossed over to Ecopipam
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Ecopipam 是多巴胺 D1 受体家族的选择性拮抗剂。
其他名称：
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Yale Global Tic Severity Scale - Total Tic Score
大体时间：Baseline and 30 days
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The Yale Global Tic Severity Scale (YGTSS) is the standard rating scale used to assess the effects of a new treatment on the symptoms of Tourette's Disorder (TD). The YGTSS is a clinician-rated, multi-dimensional instrument for assessing tic symptom severity in children and adults with TD. Both motor and vocal tics are assessed for symptom number, frequency, intensity, complexity, and interference on a 0-5 Likert scale. Scores from each dimension are totaled to reflect the severity of motor tics (range 0-25) (min of 0 to max 25), vocal tics (range 0-25) (min of 0 to max 25) and combined tics, or Total Tic Score (TTS) (range 0-50) (min of 0 to max of 50, sum of motor tics and vocal tic scores). For all scores, lower score represents better outcome. The primary outcome measure is the YGTSS-TTS (Yale Global Tic Severity Scale Total Tic Score which includes both the motor and phonic tic scores). |
Baseline and 30 days
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
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YGTSS Day 16
大体时间：Baseline and Day 16
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The Yale Global Tic Severity Scale (YGTSS) is the standard rating scale used to assess the effects of a new treatment on the symptoms of Tourette's Disorder (TD). The YGTSS is a clinician-rated, multi-dimensional instrument for assessing tic symptom severity in children and adults with TD. Both motor and vocal tics are assessed for symptom number, frequency, intensity, complexity, and interference on a 0-5 Likert scale. Scores from each dimension are totaled to reflect the severity of motor tics (range 0-25) (min of 0 to max 25), vocal tics (range 0-25) (min of 0 to max 25) and combined tics, or Total Tic Score (TTS) (range 0-50) (min of 0 to max of 50, sum of motor tics and vocal tic scores). For all scores, lower score represents better outcome. The outcome measure described is the YGTSS-TTS (Yale Global Tic Severity Scale Total Tic Score which includes both the motor and phonic tic scores) at Day 16 vs baseline. |
Baseline and Day 16
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DuPaul ADHD (Attention Deficit Hyperactivity Disorder) Rating Scale-IV
大体时间：Baseline and 30 days
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This is a validated rating scale for the symptoms of attention deficit disorder.
Scale includes 18 core items, with higher score indicating greater severity of ADHD (Total score 0-54).
These scores are compared from baseline and 30 days below; There are also subscales but not individually shown.
The subscales include 9 items measured for Inattention; 9 for Hyperactivity/Impulsivity. A score of 2 or 3 on any item is considered clinically significant.
Measure on 4-point Likert scale and also include optional performance items.
Raw scores are summed for each subscale.
Scores are compared to norm-referenced percentiles based on age and gender.
Higher scores may indicate ADHD.
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Baseline and 30 days
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Child Yale-Brown Obsessive Compulsive Scale
大体时间：Baseline and 30 days
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The CY-BOCS is a clinician-rated, 10-item scale to both determine severity of OCD and to monitor improvement during treatment, with a higher score indicating greater severity. The scale is a clinician-rated, 10-item scale (5 items regarding severity and 5 items regarding obsessions) that includes questions about the amount of time spent on obsessions/compulsions, level of impairment or distress, and how much resistance and control subjects have over these thoughts. The 10 items are assessed on a 6-point scale with an overall score. Total score from 0 to 40 with higher score indicating greater severity. Total score change shown below. |
Baseline and 30 days
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Clinical Global Impression Scale - Severity
大体时间：Baseline and 30 days
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Clinical Global Impression Scales (improvement and severity) are validated rating scales that measure whether the treatment improves the symptoms of the disease (CGI-I) and whether the treatment reduces the severity of the disease (CGI-S).
The CGI consists of two reliable and valid 7-item Likert scales used to assess severity and change in clinical symptoms.
The severity scale (CGI-S) ranges from 1 (Normal, not ill) to 7 (extremely ill).
The improvement scale (CGI-I) ranges from 1 (very much improved) to 7 (very much worse) with a score of 1 or 2 defining positive response.
The improvement rating compares the subject's overall clinical condition to the Baseline visit and the rater should consider the following question: "Compared to tic severity at baseline, how much has the subject changed?"
Data shows how much improvement at Day 30 with Ecopipam vs Placebo.
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Baseline and 30 days
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合作者和调查者
合作者
调查人员
- 学习椅：Donald Gilbert, MD、Children's Hospital Medical Center, Cincinnati
出版物和有用的链接
一般刊物
- Gilbert DL, Budman CL, Singer HS, Kurlan R, Chipkin RE. A D1 receptor antagonist, ecopipam, for treatment of tics in Tourette syndrome. Clin Neuropharmacol. 2014 Jan-Feb;37(1):26-30. doi: 10.1097/WNF.0000000000000017.
- Gilbert DL, Murphy TK, Jankovic J, Budman CL, Black KJ, Kurlan RM, Coffman KA, McCracken JT, Juncos J, Grant JE, Chipkin RE. Ecopipam, a D1 receptor antagonist, for treatment of tourette syndrome in children: A randomized, placebo-controlled crossover study. Mov Disord. 2018 Aug;33(8):1272-1280. doi: 10.1002/mds.27457. Epub 2018 Sep 7.
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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