NCT06377930已完成2 期
A Phase 2A, Multicenter, Open-label Study Evaluating the Safety and Efficacy of RAP-219 in Adult Participants With Refractory Focal Epilepsy
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 12
- 主要终点
- Change in long episode frequency during the treatment period compared to pre-treatment baseline: Responder proportion, Percent Change Percent change
研究概览
简要总结
This is a clinical research study for an investigational drug called RAP-219 in patients with Refractory Focal Epilepsy. This study is being conducted to determine if RAP-219 works and is safe in patients with Refractory Focal Epilepsy.
详细描述
This is a Phase 2A, proof-of-concept, multi-center, open-label study designed to evaluate the efficacy, safety, tolerability, and PK/PD relationship of RAP-219 in adult participants with refractory focal epilepsy. The activity of RAP-219 will be assessed in approximately 30 participants treated with the RNS® system.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Medically refractory focal epilepsy with an implanted responsive neurostimulator device placed correctly in a seizure onset zone that meet several device-related criteria and demonstrated history of compliance with interrogation of the RNS device and upload of data
- •If currently treated with antiseizure medications, up to a maximum of 4 concomitant mediations are allowed
- •At least 1 clinical seizure during the 8-week retrospective eligibility period
- •Participants in otherwise good health as determined by the investigator
- •Willing and able to adhere to all aspects of the protocol
- •A demonstrated history of compliance with interrogation of the RNS device and upload of data before screening
排除标准
- •Participants with generalized onset seizures in the past 10 years
- •History of status epilepticus while on antiseizure medications within 2 years of screening
- •Individuals of reproductive potential who do not agree to simultaneously use two effective birth-control methods
- •Participants who have had epilepsy surgery within the last 12 months before screening
研究组 & 干预措施
RAP-219
Experimental
Participants will receive 0.75 mg RAP-219 daily for 5 days followed by 1.25 mg RAP-219 daily for the remainder of the 8-week treatment period.
干预措施: RAP-219 (Drug)
结局指标
主要结局
Change in long episode frequency during the treatment period compared to pre-treatment baseline: Responder proportion, Percent Change Percent change
时间窗: Screening until 5 months after enrollment
Change in long episode frequency per 28-day period during the treatment period compared to baseline
次要结局
- Change in focal clinical seizure frequency during the treatment period compared to pre-treatment baseline: Percent Change, Responder proportion(Screening until 5 months after enrollment)
- Change in frequency of estimated electrographic seizures based on RNS® system data during the treatment period compared to pre-treatment baseline(Screening until 5 months after enrollment)
- Change in RNS® system long episode-free days during the treatment period compared to pre-treatment baseline: Count, Longest continuous interval, Time until pre-treatment count(Screening until 5 months after enrollment)
- Change in focal clinical seizure-free days during the treatment period compared to pre-treatment baseline: Count, Longest continuous interval, Time until pre-treatment count(Screening until 5 months after enrollment)
- Change in RNS® system electrographic biomarkers other than long episodes during the treatment period compared to pre-treatment baseline(Dosing until 5 months after dosing)
- Change in the Seizure Severity Response Questionnaire (SSRQ) during the treatment period compared to pre-treatment baseline(Screening until 5 months after enrollment)
- Count and proportion of responders on the clinical global impression of change (CGIC) scale during the treatment period compared to pre-treatment baseline(Screening until 5 months after enrollment)
- Plasma concentrations of RAP-219(Dosing until 5 months after dosing)
- Incidence of treatment-emergent adverse events (TEAEs)(Dosing until 5 months after enrollment)
- Clinically meaningful changes, if any, from baseline in vital signs, electrocardiograms (ECGs), complete blood counts, serum chemistries, or liver function tests after treatment with RAP-219(Screening until 5 months after enrollment)
- Incidence of participants expressing suicidal ideation assessed with the Columbia-Suicide Severity Rating Scale (C-SSRS)(Screening until 5 months after enrollment)
- Cognitive function as measured using the Cogstate Digital Battery(Screening until 5 months after enrollment)
研究者
研究点 (12)
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