Observational, Prospective, Multicenter Study of Epidyolex® (Cannabidiol CBD 100 mg/ml) Oral Solution, as Adjunctive Treatment for Seizures Associated With Lennox- Gastaut Syndrome (LGS), Dravet Syndrome (DS) and Tuberous Sclerosis Complex (TSC)
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 111
- 试验地点
- 32
- 主要终点
- Number of Participants Remaining on Therapy from Enrollment
研究概览
简要总结
This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics. The eligible participants are expected to participate in the study for a duration of 52 weeks of treatment.
详细描述
This observational study evaluates a Real-World population of children and adult participants affected by LGS, DS, and TSC and the effect of therapy with Epidyolex® administered according to clinical practice on epileptic symptoms. The study will assess specific scales, questionnaires, and the satisfaction of the caregivers and the participants/tutors.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •Adult patients, and minor patients ≥ 2 years old diagnosed with LGS, DS or TSC.
- •Clinical decision, taken by the physician, to initiate Epidyolex®
- •Adult participants, parents or legal representatives must be willing and able to give informed consent/assent for participation in the study.
排除标准
- •Participants currently using or have used recreational, medicinal cannabis, or cannabinoid-based products within the three months prior to study entry and are unwilling to abstain from these products for the duration of the study.
- •Any reason, according to Investigator's judgment, able to compromise compliance with procedures outlined in the study There will not be any other specific exclusion criteria; however, contraindications, special warnings, and precautions for use as detailed in the Summary of Product Characteristics (SmPC) (particularly related to raising of aspartate aminotransferase [AST], alanine aminotransferase [ALT], and total bilirubin) will have to be considered by the treating physician.
研究组 & 干预措施
Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex
Participants ≥2 years of age diagnosed with LGS, DS, and TSC.
干预措施: Epidiolex 100 mg/mL Oral Solution (Drug)
结局指标
主要结局
Number of Participants Remaining on Therapy from Enrollment
时间窗: Baseline up to Week 52 post-dose.
Treatment retention will be evaluated through the proportion of participants remaining on therapy from the enrollment visit (baseline, V0) to each study visit (Weeks 4 \[V1\], 16 \[V2\], 28 \[V3\], 40 \[V4\], 52 \[V5\]).
次要结局
- Average Number of Seizure-Free Days in the Last 28 Days(Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5))
- Longest Duration of Seizure Free Days in the Last 28 Days(Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5))
- Average Maintenance Dose of Epidyolex®(Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5))
- Maximum Maintenance Dose of Epidyolex®(Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5))
- Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs)(Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5))
- Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy(Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5))
- Change from Baseline to Week 28 (V3) in the Child Behavior Check List (CBCL)(Baseline to week 28 (V3) post-dose.)
- Change from Baseline to Week 52 (V5) in the Child Behavior Check List (CBCL)(Baseline to week 52 (V5) post-dose.)
- Change from Baseline to Week 28 (V3) in the Visual Analog Scale (VAS) of 10 Non-seizure Related Outcomes (NSRO)(Baseline to week 28 (V3) post-dose.)
- Change from Baseline to Week 52 (V5) in the VAS of 10 NSRO(Baseline to week 52 (V5) post-dose.)
- Average Caregiver Global Impression of Change (CGIC) Score at Week 28 (V3)(Week 28 (V3) post-dose.)
- Average CGIC Score at Week 52 (V5)(Week 52 (V5) post-dose.)
- Average Caregiver Global Impression of Change in Seizure Duration (CGICSD) Score at Week 28 (V3)(Week 28 (V3) post-dose.)
- Average CGICSD Score at Week 52 (V5)(Week 52 (V5) post-dose.)
- Average Physician Global Impression of Change (PGIC) Score at Week 28 (V3)(Week 28 (V3) post-dose.)
- Average PGIC Score at Week 52 (V5)(Week 52 (V5) post-dose.)