Sabril for Treatment of Adult Refractory Partial Seizures Symptomatic of Tuberous Sclerosis: An Open Label, Phase IV Prospective Safety and Tolerability Study
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Number of Participants Safely Tolerating Sabril
研究概览
简要总结
This is an open-label, phase 4 study to examine the safety and efficacy of vigabatrin (Sabril) in Tuberous Sclerosis patients, a subset of the larger refractory complex partial epilepsy population for which the drug is approved. While enrolled on this trial, subjects will continue to take all of their normally prescribed medications, including their other antiepileptic drugs (AEDs).
Alternatively, there is a prospective observational arm that subjects who are about to take Sabril as treatment for seizures associated with Tuberous Sclerosis may join. Subjects who join this arm will not have any study visits and will not be asked to do anything specifically for the study. The study team will collect all study data from subjects' medical records only.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient or patient's legally authorized representative must sign and date the Institutional Review Board approved Informed Consent and HIPPA Authorization Form.
- •Male and female patients 18+ years of age with a clinical diagnosis of Tuberous Sclerosis who experience an average of at least three partial seizures every two months, of which one must be a complex partial seizure
- •Patient must be on at least one and a maximum of four AEDs. Patient must be on a stable AED dose regimen for at least 30 days prior to screening. Neither a Vagal Nerve Stimulator (VNS) nor the ketogenic diet will count as an AED
- •In the investigator's opinion, the patient or caregiver must be able to keep a seizure diary
- •An MRI in the last 5 years, or willingness to undergo an MRI as part of the screening process
排除标准
- •Cause of patient's seizures is a neurologic disease that is not Tuberous Sclerosis
- •Current, clinical diagnosis of a major depressive episode or suicidal ideation
- •Patient is taking more than four concurrent AEDs. Note: VNS or ketogenic diet is allowed and will not be counted in the four allowed AEDs
- •Patient has a progressive CNS lesion confirmed by magnetic resonance imaging (MRI) or computed tomography (CT) scan
- •Patient is currently abusing drugs or alcohol
研究组 & 干预措施
Treatment with Sabril (vigabatrin)
This is a single arm study. All subjects who are eligible for treatment will begin taking vigabatrin (Sabril) during the third month of the study. Treatment will be in accordance with the FDA-approved prescribing information: upward titration will happen at a rate of 500mg per week until subjects reach their maximum tolerated dose, or 3g per day (whichever is lower). This dose may be decreased if needed under the supervision of the study doctor. Subjects who need to lower their dose or who stop taking Sabril will have their dosage decreased at a rate of 1 gm/week for one month under the supervision of the study doctor.
干预措施: vigabatrin (Drug)
结局指标
主要结局
Number of Participants Safely Tolerating Sabril
时间窗: Outcome measures will be assessed at the initiation of Sabril (titration), and at three and five months after starting Sabril. After this time, the subjects will have completed the study.
* Antiepileptic Drug (AED) levels in blood * Comprehensive panel (blood test) * Complete Blood Count with differential (blood test) * Visual field tests testing * Ophthalmology exam assessment * Frequency and severity of adverse events reported by subjects throughout their involvement with the study
次要结局
- Number of Patients Who Become Seizure Free While Taking Sabril(Seizure freedom will be assessed for the two month treatment phase of the study (months 4 and 5))