A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Participants With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder

赞助:

合作者:

信息的提供 (责任方):

，

追踪信息

首次提交日期 ^ICMJE

September 27, 2018

首次发布日期e ^ICMJE

October 3, 2018

最后更新发布日期

October 3, 2018

预计研究开始日期 ^ICMJE

September 10, 2018

预计主要完成日期

August 14, 2020 (主要结果测量的最终数据收集日期)

目前主要观察指标 ^ICMJE

Percent Change from Baseline in Motor Seizure Frequency[ Time Frame: up to 12 weeks ]

原始主要观察测量 ^ICMJE

与当前相同

目前的二级观察 ^ICMJE

Percentage of Participants Considered as Treatment Responders During the Treatment Period[ Time Frame: up to 12 weeks ]
Percent Change from Baseline in Motor Seizure Frequency During the Maintenance Period[ Time Frame: up to 8 weeks ]
Percent Change from Baseline in Clinician's Global Impression of Severity and Change (CGI-S/C) Responses of Investigator[ Time Frame: up to 12 weeks ]
Correlation of TAK-935 Concentration and Plasma 24S-hydroxycholesterol (24HC) Levels[ Time Frame: up to 12 weeks ]
Percent Change from Baseline in Care Clinical Global Impression of Change (CGI-C) Responses of Parent/Family[ Time Frame: up to 12 weeks ]

描述性信息

简略标题 ^ICMJE

A Multicenter, Open-label, Pilot Study of TAK-935 (OV935) in Participants With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder

正式标题 ^ICMJE

A Multicenter, Open-label, Pilot Study of TAK-935 in Patients With 15Q Duplication Syndrome or CDKL5 Deficiency Disorder

简要概况

The purpose of this study is to investigate the effect of TAK-935 (OV935) on the frequency of motor seizures for participants with 15q duplication syndrome or CDKL5 deficiency disorder throughout the Treatment Period (Titration and Maintenance).

详细说明

The drug being tested in this study is called TAK-935 (OV935). TAK-935 is being tested to treat people with 15q duplication syndrome or CDKL5 deficiency disorder. This study will assess the effects of TAK-935 on seizure frequency, safety. The study will enroll approximately 30 participants. Participants will be enrolled into 2 groups based on their diagnosis as: 15q duplication syndrome or CDKL5 deficiency disorder. All participants will be asked to take TAK-935 tablets twice daily with or without food. The study comprises of 2 periods: Screening/Baseline Period and Treatment Period. The overall time to participate in this study is 22 weeks, including 4 to 6 weeks Screening/Baseline Period, 12 weeks Treatment Period, 2 weeks Taper, and 2 weeks safety follow up period. Participants completing this study will have an option to enroll in the open-label extension (OLE) study, under a separate protocol.

研究类型 ^ICMJE

Interventional

研究阶段

Phase 2

研究设计 ^ICMJE

分配:
干预模型: Single Group Assignment
干预模型描述:
盲法: Interventional
盲法描述:
主要目的: Treatment

适用条件 ^ICMJE

干预项目 ^ICMJE

Drug: TAK-935
TAK-935 tablets or mini-tablets.

研究工具

Experimental: TAK-935 (OV935)
Treatment: 2 weeks titration followed by 10 weeks maintenance period.

招募信息

招募状态 ^ICMJE

Recruiting

预计入组 ^ICMJE

原始预计入组 ^ICMJE

与当前相同

预计研究完成日期

September 11, 2020

预计主要完成日期

August 14, 2020 (主要结果测量的最终数据收集日期)

合格标准 ^ICMJE

Inclusion Criteria: 1. Clinical diagnosis of 15q duplication syndrome or CDKL5 deficiency disorder 2. Currently taking 1 to 4 antiepileptic drugs (AEDs) at a stable dose Exclusion Criteria: 1. One or more episodes of convulsive status epilepticus per week requiring hospitalization 2. Currently receiving a study drug or participated in a clinical study involving another investigational product in the previous month

性别

参与研究的性别:

All

年龄

最小年龄：2 Years ，最大年龄：17 Years

接受健康的志愿者

没有

可入组国家 ^ICMJE

United States

管理信息

数据检测委员会

Yes

研究涉及美国FDA监管的产品

研究美国FDA监管的药品: No
研究涉及美国FDA监管的设备产品: No

IPD 共享声明

计划分享 IPD:

Undecided

责任方

，

研究赞助商 ^ICMJE

Takeda

合作者 ^ICMJE

研究员 ^ICMJE

Study Director: Medical Director Takeda

PRS 账户

验证日期

October 2018

^ICMJE 国际医学期刊编辑委员会和世界卫生组织 ICTRP 要求的元素

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