Efficacy of Ketone Esters for Children With Drug Resistant Epilepsy
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- ≥ 50% reduction in seizure frequency
研究概览
简要总结
This study aims to investigate the efficacy of add-on exogenous ketone esters for treating children with drug-resistant epilepsy
详细描述
Epilepsy is a common neurological disorder among children with significant neurobiological, cognitive, psychological, and social consequences. Seizures can usually be controlled by anti-seizure medications (ASMs) in up to two-thirds of children with epilepsy. However, this leaves a significant part of epileptic children whose seizures are not controlled by pharmacotherapy. Currently, available alternatives for drug-resistant epilepsy (DRE) include surgery, vagus nerve stimulation, and ketogenic diet (KD).
KD has been classically used for treating children with DRE. However, KD requires strict dietary restriction, which may not be applicable or acceptable for many patients, and is associated with several adverse effects, commonly including gastrointestinal (e.g., constipation, nausea, vomiting), cardiovascular (e.g., dyslipidemia), renal/genitourinary (e.g., renal calculi), and growth problems. Exogenous ketone esters (EKE) could be a more convenient and superior alternative to KD for children with DRE.
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
- None
入排标准
- 年龄范围
- 1 Year 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Drug-resistant epilepsy
- •Seizure frequency ≥ 7 per week
排除标准
- •Failure to obtain informed consent
- •Recent intake of exogenous ketones, ketogenic diet, or any dietary restrictions/modifications
- •Severe disease conditions, including hepatic, renal, respiratory, cardiac, gastrointestinal, endocrinal, and immune systems
- •Hypo-/hyperglycemia
- •Metabolic acidosis
- •Ketosis (βHB > 2 mmol/L)
- •GIT disorders, including gastritis/peptic ulcer, diarrhea/constipation, and irritable bowel disease
- •Malnutrition/obesity
- •Limitations to oral feeding (e.g., severe gastroesophageal reflux)
- •Inborn errors of metabolism
- •Chromosomal disorders
- •Surgically-remediable epilepsy
- •Allergies or any other contraindication to ketone supplements
- •Inapplicable recording of seizures
- •Incompliance to anti-seizure medications and/or irregular follow-up
- •Recent propofol therapy
- •Intake of carbonic-anhydrase inhibitors
研究组 & 干预措施
Study group
Children receiving exogenous ketone esters + standard of care
干预措施: Exogenous ketone ester (Drug)
结局指标
主要结局
≥ 50% reduction in seizure frequency
时间窗: From 28-days observation (baseline) phase to 28-days intervention phase
Proportion of patients achieving ≥ 50% reduction in seizure frequency
次要结局
- Proportion of incompliance to exogenous ketone ester therapy(28-days intervention phase)
- Proportion of incompliance to anti-seizure medications (ASMs)(From 28-days observation (baseline) phase to 28-days intervention phase)
- Change in seizure severity assessed by National Hospital Seizure Severity Scale (NHS3)(From 28-days observation (baseline) phase to 28-days intervention phase)
- Change in cognitive domains(From 28-days observation (baseline) phase to 28-days intervention phase)
- Change in frequency of status epilepticus(From 28-days observation (baseline) phase to 28-days intervention phase)
- Change in seizure frequency(From 28-days observation (baseline) phase to 28-days intervention phase)
- Change in blood βHB(From baseline to 30 minutes, 1 hour, 2 hours, 4 hours, 2 days, 4 days, 7 days, 14 days, and 28 days study timepoints)
- Change in occurrence of possible adverse effects(From 28-days observation (baseline) phase to 28-days intervention phase)
- Change in blood glucose(From baseline to 30 minutes, 1 hour, 2 hours, 4 hours, 2 days, 4 days, 7 days, 14 days, and 28 days study timepoints)
- Change in blood pH(From baseline to 30 minutes, 1 hour, 2 hours, 4 hours, 2 days, 4 days, 7 days, 14 days, and 28 days study timepoints)
- Change in EEG score(From baseline to 28 days study timepoint)
研究者
Elsayed Abdelkreem
Lecturer of Pediatrics
Sohag University