NCT00357669已完成3 期
A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Study to Evaluate the Efficacy and Safety of Brivaracetam Used as Adjunctive Treatment for 12 Weeks in Adolescent and Adult Patients (≥16 Years) With Genetically Ascertained Unverricht-Lundborg Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 50
- 主要终点
- Percent reduction from baseline on the Action Myoclonus score (Unified Myoclonus Rating Scale (UMRS) Section 4) at the end of the Treatment Period
研究概览
简要总结
The study will compare the efficacy and safety of brivaracetam with placebo in patients with Unverricht-Lundborg disease.
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
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects with diagnosed Unverricht-Lundborg disease (ULD) ascertained by appropriate genetic testing for a homozygous or compound heterozygous mutation in the Cystatin B (CSTB) gene
- •Subjects with moderate to severe myoclonus documented by an Action Myoclonus sum score of ≥ 30 (evaluation by investigator)
- •Subjects currently being or having been treated with clonazepam up to the maximum recommended daily dose of 20 mg or up to their individual optimal dose as assessed by the investigator
- •Subjects currently being or having been treated with valproate up to the maximum recommended daily dose 60 mg/kg or serum levels of 100 mcg/ml or up to their individual optimal dose as specified by the investigator
排除标准
- •Subjects currently on felbamate or having been on felbamate within less than 18 months prior to Visit 1
- •Subjects currently treated with phenytoin or having been on phenytoin in the last month prior to Visit 1
- •Subjects currently on vigabatrine. Subjects having been on vigabatrine if no visual fields examination report available including standard static (Humphrey or Octopus) or cinetic perimetry (Goldman)
- •Subject taking any drug with possible central nervous system (CNS) effects
- •Subjects taking any drug that may significantly influence the metabolism of BRV (CYP2C or CYP3A potent inducers/inhibitors)
- •Known clinically significant acute or chronic illness or illness which may impair reliable participation in the trial, necessitate the use of medication not allowed by protocol or represent a safety risk in the Investigator's opinion
- •Subjects with history of severe adverse hematological reaction to any drug
- •Impaired hepatic function: ALAT/SGPT, ASAT/SGOT, alkaline phosphatase, GGT value of more than three times the upper limit of the reference range
- •History of suicide attempt during the last 5 years
- •Subject with suicidal ideations within the last year or at risk of suicide attempt unless cleared by written confirmation from a psychiatrist and approved by the UCB physician
- •Ongoing psychiatric disorder other than mild controlled disorder
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Placebo (Other)
Brivaracetam 50 mg/day
Experimental
BRV 50 mg/day
干预措施: Brivaracetam 25 mg (Drug)
Brivaracetam 50 mg/day
Experimental
BRV 50 mg/day
干预措施: Brivaracetam 50 mg (Drug)
Brivaracetam 150 mg/day
Experimental
BRV 150 mg/day
干预措施: Brivaracetam 25 mg (Drug)
Brivaracetam 150 mg/day
Experimental
BRV 150 mg/day
干预措施: Brivaracetam 50 mg (Drug)
结局指标
主要结局
Percent reduction from baseline on the Action Myoclonus score (Unified Myoclonus Rating Scale (UMRS) Section 4) at the end of the Treatment Period
时间窗: End of treatment period (Week 14 or early discontinuation visit)
次要结局
- Percent reduction from baseline on the functional disability score (UMRS Section 5) at the end of the Treatment Period(End of treatment period (week 14 or early discontinuation visit))
- Percent reduction from baseline on the stimulus sensitivity score (UMRS Section 3) at the end of the Treatment Period(End of treatment period (week 14 or early discontinuation visit))
- Percent reduction from baseline on the myoclonus patient questionnaire (UMRS Section 1) at the end of the Treatment Period(End of treatment period (week 14 or early discontinuation visit))
- Global Evaluation Scale by Investigator (I-GES) at the end of the Treatment Period(End of treatment period (week 14 or early discontinuation visit))
研究者
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