A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy Followed by Long-term Open-label Treatment.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 29
- 试验地点
- 1
- 主要终点
- Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)
研究概览
简要总结
A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in female children and young adults with genetically confirmed PCDH19 gene mutation.
详细描述
The Violet Study is a global, double-blind, placebo-controlled, Phase 2 clinical trial that plans to enroll approximately 25 female patients between the ages of 1 and 17 with a confirmed disease-related PCDH19 gene variant. Patients will undergo a baseline period before being randomized to receive, in addition to their existing anti-seizure treatment, either ganaxolone or placebo for 17 weeks. Following the treatment period, all patients that meet certain eligibility requirements will have the opportunity to receive ganaxolone in the open label phase of the study. The study's primary efficacy endpoint is percent reduction in seizures. Secondary outcome measures will include non-seizure-related endpoints to capture certain behavioral and sleep disturbances that have been seen in previous clinical studies with ganaxolone.
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
- 1 Year 至 17 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Molecular confirmation of a pathogenic or likely pathogenic PCDH19 variant
- •Failure to control seizures despite 2 or more anti-seizure medications
- •12 seizures over a 12-week period of primary seizure types prior to screening
- •On a stable regimen of concomitant AEDs, Ketogenic diets, and modified Atkins diet should be unchanged for 3 months prior to screening)
排除标准
- •Previous exposure to ganaxolone
- •> 8 consecutive weeks of seizure freedom during the 12 weeks prior to screening
- •Concurrent use of strong inducers or inhibitors of CYP3A4/5/7 is not permitted
- •Use of tetrahydrocannabinol (THC) or non-approved cannabidiol (CBD) is prohibited during the double-blind phase
- •Exposure to any other investigational drug within 30 days or fewer than 5 half-lives prior to screening
研究组 & 干预措施
Placebo
placebo suspension 3x's /day for 17 weeks
干预措施: Placebo (Drug)
Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
干预措施: Ganaxolone (Drug)
结局指标
主要结局
Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)
时间窗: End of the double-blind 17 week treatment period
Summary of 28-Day Seizure Frequency for Seizure Types through 17 week Post-Baseline Phase (Median Percent Change)
次要结局
- Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change)([Time Frame: End of the double-blind 17 week treatment period])
- 50% Primary Seizure Reduction(End of the double-blind 17 week treatment period)