Open Label, Single-Arm Study of the Safety and Tolerability of a Leucine Enriched Essential Amino Acid Powder as an Add-on to a Classic Ketogenic Diet in Refractory Epilepsy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Number of participants with treatment-related adverse events
研究概览
简要总结
The main purpose of this study is to evaluate the safety and tolerability of a Leucine-Enriched Essential Amino Acid (LEAA) Powder as an add-on to a classic ketogenic diet (KD) in pediatric and adult patients with refractory epilepsy.
详细描述
This is a single center, single-arm, open label study to evaluate the safety, tolerability, and nutritional effect of Leucine-Enriched Essential Amino Acid powder (LEAA) when added to classical KD in pediatric and adult patients whose epilepsy is insufficiently controlled on the diet. The study will enroll patients who have previously been compliant with their classical KD regimen for at least 28 days prior to enrollment and have experienced some response (≥40% reduction in seizures), but not complete response (<90% reduction in seizures) to dietary management with KD. Study duration is 84 days and will have 3 remote study visits (Baseline- Remote Visit 1, Dose Adjustment- Remote Visit 2, and Final- Remote Visit 3) and a pre-screening communication via telephone/video.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients ages 2 years and up at enrollment.
- •A diagnosis of epilepsy, with at least 2 seizures per week according to the parent/guardian report to study staff and investigator medical notes if applicable. All seizure types allowed.
- •Under stable treatment with a classic ketogenic diet (KD) regimen with a KD ratio ≥ 1:1, with or without antiseizure medications (ASMs), for at least 28 days with documented, partial response (≥40% reduction in seizures), but not a complete response (<90% reduction in seizures) based on patient medical records or parental report.
- •Patient and/or parent/caregiver able and willing to follow all study procedures and successfully complete the study.
排除标准
- •Patients who have changed their KD regimen or antiseizure medication (ASM) or vagus nerve stimulation (VNS) within the last 28 days.
- •Allergy, sensitivity to, or inability to metabolize any component of leucine enriched essential amino acid (LEAA).
- •Those who are pregnant or breastfeeding.
- •Patients who have received an investigational drug or product or participated in a drug study within 4 weeks before the first dose of leucine enriched essential amino acid (LEAA) powder.
- •Patients who have a clinically significant condition or have had either clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to the Baseline Visit 1, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the patient including liver disease.
- •Patients with history of suicidal ideation in past 6-months and suicidal behavior in past 2-years
研究组 & 干预措施
Leucine-Enriched Essential Amino Acid powder (LEAA)
Leucine-Enriched Essential Amino Acid powder (LEAA) as an add-on to a classic ketogenic diet (KD)
干预措施: Leucine-Enriched Essential Amino Acid powder (Drug)
结局指标
主要结局
Number of participants with treatment-related adverse events
时间窗: Baseline, 3 weeks, 12 weeks
Number of participants with treatment-related adverse events as reported by the trial participants.
次要结局
- Number of participants with improved nutritional status as assessed by serum ketone and glucose level.(At each visit and on next day)
- Number of participants with improved nutritional status as assessed by weight.(Baseline, 3 weeks, 12 weeks)
- Number of participants with improved nutritional status as assessed by body mass index (BMI).(Baseline, 3 weeks, 12 weeks)
研究者
Elizabeth Anne Thiele
Director, Pediatric epilepsy program, Director, Herscot Center for TSC, Professor of Neurology, Harvard Medical School
Massachusetts General Hospital