Pilot Randomized, Placebo-Controlled Trial to Evaluate The Effect of Oral Pulsed Methylprednisolone on Seizure Frequency in Pediatric Patients With Idiopathic Intractable Convulsive Epilepsy
试验速览
- 阶段
- 1 期
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Seizure frequency
研究概览
简要总结
Although corticosteroids have been shown to be beneficial anecdotally for refractory epilepsy, the effects of corticosteroids on pediatric epilepsy have primarily been studied retrospectively amongst a heterogeneous patient population. The objective of this prospective cross-over study is to determine the effect of oral steroids on convulsive seizure frequency and evaluate the tolerability of pulsed oral steroids. Participants will be prospectively enrolled from pediatric neurology clinic at Children's Hospital of New Orleans, and baseline seizure frequency will be assessed. Participants will then be randomized to receive either the study drug, methylprednisolone, or placebo during the first month, followed by a one-month wash-out period. During the third month of the study, participants will enter the cross-over phase of the study where those who received methylprednisolone will receive placebo, and those who received placebo will receive methylprednisolone. The primary outcome will be the percentage of patients with 50% or more reduction in seizure frequency following one course of oral methylprednisolone. frequency following 1 course of oral methylprednisolone.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients age 2 -18 years of age
- •Patients who have at least 4 convulsive (generalized tonic or tonic-clonic) seizures per month on 2 or more anti-epileptic drugs (AEDs) at therapeutic doses
- •a. Epilepsy diagnosed by historical clinical evidence
- •Family's ability to understand and willingness to sign a written informed consent document for patients under
- •Willingness to complete seizure diary for duration of study
- •Willingness to present to all study visits
排除标准
- •Patients with history of the following diagnoses:
- •Traumatic brain injury
- •Tuberous sclerosis
- •Sturge Weber
- •Cortical dysplasia
- •Patients with known hereditary degenerative diseases as follows:
- •Adrenoleukodystrophy
- •Neuronal ceroid lipofuscinosis
- •Leigh Syndrome
- •Myoclonic epilepsy with ragged red fibers (MERRF)
- •Rett Syndrome
- •Patients with the following epilepsy syndromes
- •Infantile spasms
- •West Syndrome
- •Progressive myoclonic epilepsy
- •Dravet syndrome
- •Doose syndrome
- •Ohtahara syndrome
- •Rasmussen's encephalitis
- •Patients with the following metabolic disorders
- •Phenylketonuria
- •Maple syrup urine disease
- •Organic acidemias
- •Galactosemia
- •Peroxismal disorders (e.g. Zellwegers)
- •Lysosomal disorders
- •Urea cycle disorders
- •Patients with history of immunodeficiency
- •Patients with the following infections
- •Active or latent TB
- •Active or suspected bacterial infection
- •Active, latent or suspected fungemia
- •Active or suspected parasitic infection
- •Patients with history of malignancy
- •Patients with history of or active myopathy
- •Patients with degenerative neuromuscular disorders
- •Patients with history of hypersensitivity or allergic reactions to corticosteroids
- •Patients with history of psychosis
- •Patients with diabetes mellitus
- •Any underlying predisposition to gastrointestinal bleeding (peptic ulcer disease, gastritis, colitis)
研究组 & 干预措施
Interventional start
Patients who randomize to the interventional start arm will receive the study drug, methylprednisolone sodium succinate, in the first month of the study, followed by placebo in the cross-over phase of the study.
干预措施: methylprednisolone sodium succinate (Drug)
Interventional start
Patients who randomize to the interventional start arm will receive the study drug, methylprednisolone sodium succinate, in the first month of the study, followed by placebo in the cross-over phase of the study.
干预措施: Placebo (Other)
Placebo start
Patients who randomize to the placebo start arm will receive placebo in the first month of the study, followed by the study drug, methylprednisolone sodium succinate, in the cross-over phase of the study.
干预措施: methylprednisolone sodium succinate (Drug)
Placebo start
Patients who randomize to the placebo start arm will receive placebo in the first month of the study, followed by the study drug, methylprednisolone sodium succinate, in the cross-over phase of the study.
干预措施: Placebo (Other)
结局指标
主要结局
Seizure frequency
时间窗: The number of seizures per month will be assessed at 1 month (following placebo/intervention), 2 months (following wash-out), and 3 months (following cross-over phase of placebo/intervention). Change compared to baseline will be calculated.
The percentage of patients with 50% or more reduction in seizure frequency.
次要结局
- Seizure freedom(The number of seizures per month will be assessed at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).)
- Adverse events(Adverse events will be recorded at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).)
- Drop out percentage(The number of participants who drop out will be counted and recorded at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).)