Multicentre, Randomized, Controlled Clinical Trial on 7-day Followed by Maintenance Therapy for 10 Weeks Vs. 14-day and No Maintenance Course of Prednisolone for the Treatment of Infantile Epileptic Spasms Syndrome (IESS)
试验速览
- 阶段
- 4 期
- 状态
- 进行中（未招募）
- 入组人数
- 182
- 试验地点
- 7
- 主要终点
- Electroclinical Remission at Day 14 and Day 28
研究概览
简要总结
The goal of this clinical trial is to learn if short courses (7 days) of oral prednisolone are as effective as longer courses (14 days) in treating Infantile Epileptic Spasms Syndrome (IESS) in infants. The main questions it aims to answer are:
- Does a 7-day course of oral prednisolone result in a similar or better reduction in spasm frequency compared to a 14-day course?
- Does the duration of treatment (7 vs. 14 days) influence relapse rates and developmental outcomes in infants with IESS?
- Researchers will compare the effects of the two treatment arms (7-day course vs. 14-day course of oral prednisolone) to see if there is a difference in efficacy and safety.
Participants will:
- Receive either a 7-day or 14-day course of oral prednisolone as part of their treatment
- Be monitored for spasm frequency and any side effects during hospital observation for the first 48 hours
- Maintain a spasm diary during the treatment period to track spasm frequency
- Return for follow-up visits at 7 days, 14 days, 28 days, 42 days, 3 months, 6 months, and 12 months to assess treatment response, relapse, and developmental outcomes
详细描述
Study Title: Comparison of Short (7-Day) versus Long (14-Day) Courses of Oral Prednisolone in the Treatment of Infantile Epileptic Spasms Syndrome (IESS)
Study Overview: This is a clinical trial designed to compare the efficacy and safety of two treatment regimens using oral prednisolone for infants with Infantile Epileptic Spasms Syndrome (IESS). The study aims to evaluate the impact of treatment duration (7 days vs. 14 days) on spasm resolution, relapse rates, and developmental outcomes in infants with IESS. Participants will be randomized into two treatment arms to receive either a 7-day or 14-day course of oral prednisolone, and they will be followed for a year to assess outcomes related to spasm control, relapse, adverse effects, and long-term development.
Study Population: The study will include infants diagnosed with IESS, aged 3 to 24 months, who are receiving care at participating clinical sites. Eligibility criteria ensure the inclusion of infants with confirmed spasms, with or without known aetiology, and those who have not previously received systemic treatment for IESS.
Objectives:
- Primary Objective: To compare the effectiveness of 7-day versus 14-day oral prednisolone treatment in reducing spasm frequency in infants with IESS.
- Secondary Objectives: To evaluate the impact of treatment duration on relapse rates, developmental outcomes, and adverse effects.
- Exploratory Objectives: To assess any differences in long-term neurodevelopmental progress, including cognitive and motor development, and to compare EEG findings between treatment groups.
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
- Single (Outcomes Assessor)
盲法说明
Both the outcome assessor (MBBS-qualified research assistant) and the data analyst will be blinded to the treatment arms. By implementing these measures, the risk of bias is minimized, and the credibility of the study results is enhanced, ensuring that the conclusions drawn from the data are based solely on the observed effects of the treatments without any influence from knowledge of the treatment allocations.
入排标准
- 年龄范围
- 3 Months 至 24 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Infants between ages of 3 to 24 months
- •Newly confirmed diagnosis of infantile epileptic spasms syndrome made by the referring paediatrician/ paediatric neurologist based on the diagnostic criteria outlined in the recent ILAE classification and definition of epilepsy syndrome with onset in neonates and infants in 2022
- •Hypsarrhythmia recorded on pre-treatment EEG. This will be those who show a BASED score of 4 or 5 in a standard EEG
排除标准
- •Infants with tuberous sclerosis complex
- •Ever treated previously for infantile epileptic spasms syndrome with steroids or other anticonvulsants
- •Infants already on steroids or ACTH for any other illness
- •Contraindication for the use of high dose steroids such as underlying infection, immune deficiency, hypertension etc.
- •Children in critical conditions such as severe infections, congenital heart disease or requiring ventilation or care in an ICU
- •Not accompanied by parent/s or parent's inability to complete follow up
研究组 & 干预措施
treatment arm A
The initial phase of the intervention involves administering prednisolone orally at a dosage of 10 mg, four times daily (40 mg/day) for 7 days. This will be followed by a tapering regimen over 15 days (~2 weeks) as outlined below:
10 mg three times daily - 5 days 10 mg twice daily - 5 days 10 mg daily - 5 days The second phase of the intervention begins at the end of 15 days, where a prolonged tapering of prednisolone at a low dose (<0.4 mg/kg per day) will be administered as 10 mg once daily, twice a week, for an additional 10 weeks.
干预措施: Short-Duration Oral Prednisolone with Extended Tapering (Drug)
treatment arm B
Prednisone will be administered orally at a dose of 10 mg, four times daily (40 mg/day) for 14 days. This is the standard therapy currently given to children with IESS in Sri Lanka. This will be followed by a tapering regimen over 15 days (~2 weeks) as outlined below:
10 mg three times daily - 5 days 10 mg twice daily - 5 days 10 mg daily - 5 days No additional tapering will be implemented.
干预措施: Standard Oral Prednisolone Regimen with Tapering (Drug)
结局指标
主要结局
Electroclinical Remission at Day 14 and Day 28
时间窗: Assessed at 14 days and 28 days after treatment initiation.
This primary outcome measure compares the efficacy of the 7-day versus 14-day oral prednisolone regimens. Electroclinical remission is defined as (1) spasm control-no clinically recognizable spasms for a minimum of 24 consecutive hours as recorded by the caregiver-and (2) EEG remission-disappearance of hypsarrhythmia with the BASED score improved to 3 or less on a standardized 30-minute sleep EEG (with 5 minutes of wakefulness).
次要结局
- Long-Term Spasm Control(Assessed at 42 days, 3 months, 6 months, and 12 months post-treatment.)
- Spasm Relapse Rate and Time to Relapse(Evaluated continuously over the 12-month follow-up period.)
- Developmental Outcome at 24 Months(Assessed at 24 months following treatment initiation.)
- Evolution of Epilepsy(Assessed at 12 months post-treatment.)
研究者
Jithangi Wanigasinghe
Professor in Paediatric Neurology, Department of Paediatrics, Faculty of Medicine, University of Colombo, Sri Lanka
University of Colombo