NCT02987114已完成2 期
A Phase II, Open-label, Single-center Clinical Study to Evaluate the Safety, Tolerability and Efficacy of Oral Administration of PTL101 (Cannabidiol) as an Adjunctive Treatment for Pediatric Intractable Epilepsy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Incidence of study treatment related adverse events (AEs)
研究概览
简要总结
To evaluate the safety, tolerability and efficacy of oral administration of PTL101 (cannabidiol) for the treatment for pediatric intractable epilepsy.
Study will include a 4-week observation period ,12 weeks of treatment and 2 weeks of follow up.
详细描述
This is an open-label, single-center study recruiting approximately 15 male or female pediatric (ages 2-15, inclusive) patients with, intractable epilepsy, on stable doses of antiepileptic drugs (AEDs). The study comprised of the following period: 4 weeks observation period, followed by a 2-week dose titration period, 10-week maintenance treatment period, and a 2-week follow-up of which 1 week is a tapering-off period. Seizures will be recorded by the legal guardian/caregiver in seizure diaries throughout the first 16 weeks of the study.
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 15 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pediatric subjects with refractory epilepsy
- •2-15 years old (inclusive), male or female
- •History of the any of the following seizure types: tonic, clonic, tonic-clonic in the form of partial seizures, partial seizures secondarily generalized or primary generalized, complex partial seizures and drop attacks (tonic/atonic).
- •At least four clinically countable seizures within 4 weeks of study entry [tonic, clonic, tonic-clonic in the form of partial seizures, partial seizures secondarily generalized or primary generalized and/or complex partial seizures and drop attacks (tonic/atonic)]
- •Subject on a stable regimen of 1-4 concomitant antiepileptic drugs (AEDs) for a minimum of 4 weeks prior to enrollment
- •History of treatment with at least four AEDs, including one trial of a combination of two concomitant drugs, without successful seizure control
- •Subjects with vagal nerve stimulation system must be on stable settings for a minimum of 6 months prior to enrollment
- •For subjects undergoing dietary treatment (e.g., ketogenic or modified Atkins diet):
- •the fat to carbohydrate ratio must be stable for a minimum of eight weeks prior to enrollment
- •The subject's legal guardian voluntarily provides consent for participation in the study and signs an Informed Consent Form
- •Completed seizures diary for four weeks (±3 days) prior to initiation of the dose titration period (visit 2). Subject will be considered a screen failure if seizures diary was not appropriately completed.
排除标准
- •The subject is currently using or has used cannabis-based or synthetic cannabinoid within three months of study entry
- •Subject is unwilling to abstain from use of cannabis-based or synthetic cannabinoid throughout the study period
- •Neurodegenerative or deteriorated neurological disease
- •History of heart failure
- •Known family history (first-degree) of psychiatric disorders
- •Psychosis or past psychotic event and/or anxiety disorder
- •Current or history of drug abuse/addiction
- •Renal, hepatic (ALT/AST >2x upper limit of normal (ULN), bilirubin >2x ULN), pancreatic dysfunctions or laboratory test abnormalities, at the investigator's discretion
- •Clinically significant finding in baseline ECG
- •Initiation of felbamate treatment within 9 months of screening
- •Allergy to CBD or any cannabinoid and/or formulation excipients
- •Subject is pregnant, lactating, or planning a pregnancy during the course of the study or within 3 months of study completion
- •Subject and legal guardian/caregiver unable to comply with study visits/requirements
- •Subject is currently enrolled in, or has not yet completed a period of at least 60 days since ending another investigational device or drug trial(s)
研究组 & 干预措施
PLT101
Experimental
PTL101 capsules (50 or 100 mg CBD per capsule) up to 25 mg/kg/day or up to 450 mg/day, the lower of the two. Twice daily (morning and evening).
干预措施: PLT101 (Drug)
结局指标
主要结局
Incidence of study treatment related adverse events (AEs)
时间窗: 12 weeks of treatment + 2 weeks follow up
Percent change in mean countable monthly seizure frequency
时间窗: 12 week treatment period
次要结局
- assessment of Caregiver Global Impression of Improvement using a 5-point rating scale(after 5 weeks of maintenance dose and at end of treatment)
- Incidence of all adverse events (AEs)(12 weeks of treatment + 2 weeks follow up)
- assessment of Caregiver Global Impression of Seizure Severity using a 5-point rating scale(after 5 weeks of maintenance dose and at end of treatment)
研究者
研究点 (1)
Loading locations...
相似试验
终止
2 期
Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence SeizuresChildhood Absence EpilepsyNCT03336242Radius Pharmaceuticals, Inc.20
招募中
1 期
A Study of TY-1091 in Patients With Advanced Solid TumorsRET-altered Non Small Cell Lung CancerMedullary Thyroid CancerRET-altered Papillary Thyroid CancerNeoplasmsNCT05675605TYK Medicines, Inc248
Unknown
2 期
Evaluation of the Safety, Tolerability, and Efficacy of Orally Administered PTL201 in MS Patients With Spasticity-related SymptomsMultiple SclerosisNCT03005119PhytoTech Therapeutics, Ltd.70
终止
1 期
A Study to Evaluate VIP152 in Subjects With Relapsed/Refractory Chronic Lymphocytic Leukemia or Richter SyndromeRelapsed Non Hodgkin LymphomaRefractory Chronic Lymphocytic LeukemiaRichter SyndromeMYC AmplificationMYC OverexpressionMYC TranslocationChronic Lymphocytic LeukemiaNCT04978779Vincerx Pharma, Inc.6
已完成
2 期
An Open-Label, Single Dose Study in Patients With Alcohol Use DisorderAlcohol Use DisorderNCT05674929Beckley Psytech Limited13