A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Cannabidiol Oral Solution (CBD-OS, JZP926-OS) in Participants Aged 1 Year and Older With Developmental and Epileptic Encephalopathy (DEE)
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 120
- 主要终点
- Change in Countable Motor Seizure Frequency Per 28 Days
研究概览
简要总结
The efficacy, safety, and tolerability of CBD-OS have been evaluated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and Tuberous sclerosis complex (TSC). The current JZP926-303 study is being conducted to evaluate the safety and efficacy of CBD-OS in participants with Developmental and Epileptic Encephalopathy (DEE).
详细描述
This Phase 3, multicenter, randomized, placebo-controlled, double-blind study will evaluate the efficacy and safety of CBD-OS in participants aged ≥ 1 year with DEE. The primary objective of the 6-week Double-blind Treatment Period of the study is to assess the efficacy of CBD-OS in reducing the frequency of countable motor seizures compared with placebo in participants with DEE. In addition, the Double-Blind Treatment Period will also assess the safety and tolerability of CBD-OS. The optional 6-month open-label extension (OLE) will provide additional data on the long-term efficacy, safety, and tolerability of CBD-OS.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants are eligible to be included in the study only if all the following criteria apply:
- •Is at least 1 year of age at the time of signing the informed consent/assent.
- •Meets the clinical phenotype for DEE as specified in the protocol.
- •Per the investigator, the underlying etiology contributes to developmental impairment and seizures.
- •Has had, or is willing to complete, confirmatory imaging and/or genetic testing to determine etiology of DEE.
- •Is currently receiving antiseizure intervention, such as treatment with a stable regimen of at least 1 ASM or an established intervention for epilepsy (eg, ketogenic diet or neurostimulation).
- •All medications or interventions for epilepsy have been stable for ≥ 28 days prior to starting the baseline period (Visit 2) with no planned changes to the regimen for the duration of the Double-blind Treatment Period.
- •Participants are excluded from the study if any of the following criteria apply:
- •Has a concurrent, confirmed diagnosis of non-epileptic seizures or events that can confound the assessment of the efficacy measures, in the opinion of the investigator.
- •The etiology of the participant's seizures is a progressive neurologic disease.
- •Has known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention, such as sesame oil.
- •Has an active central nervous system (CNS) infection, demyelinating disease, degenerative neurologic disease, or any CNS disease deemed to be progressive during the study that may confound the interpretation of the study results (including autoimmune encephalitis).
- •Is currently being treated with Epidiolex or recently received treatment with Epidiolex within 28 days prior to screening.
- •Has experienced a lack of efficacy and/or poor tolerability to an adequate treatment regimen of Epidiolex based on medical history and the clinical judgement of the investigator. Participants who discontinued treatment for reasons other than safety, tolerability, or lack of efficacy and previously received Epidiolex ≥ 28 days prior to starting the Baseline Period (Visit 2) may be eligible for the study after consultation with the medical monitor and/or sponsor representative.
- •Has been taking felbamate for less than 12 months prior to screening. Participants who are stable on felbamate for ≥ 12 months are eligible for inclusion.
排除标准
- 未提供
研究组 & 干预措施
Placebo
Participants with DEE will be randomized to matching placebo for a 6-week treatment period.
干预措施: Placebo (Drug)
CBD-OS
Participants with DEE will be randomized to CBD-OS up to 10 mg/kg twice daily for a 6-week treatment period.
干预措施: CBD-OS (Drug)
Open-Label Extension: CBD-OS
Participants with DEE who completed the double-blind phase of the study and enter the optional OLE will begin a 22-week open-label treatment period with CBD-OS up to 10 mg/kg twice daily following a 2-week Blinded Transition Period.
干预措施: CBD-OS (Drug)
结局指标
主要结局
Change in Countable Motor Seizure Frequency Per 28 Days
时间窗: Baseline up to 6 weeks of double-blind treatment period
次要结局
- Change in Total Seizure Frequency Per 28 Days(Baseline up to 6 weeks of double-blind treatment period)
- Proportion of Participants Who Achieve ≥ 50% Reduction From Baseline in Countable Motor Seizure Frequency(Baseline up to 6 weeks of double-blind treatment period)
- Caregiver Global Impression of Change (CaGI-C) Score(Week 6 of double-blind treatment period)
- Change from Baseline in Caregiver Global Impression of Severity (CaGI-S) Score(Week 6 of double-blind treatment period)
- Change From Baseline in Number of Countable Motor Seizure-free Days per 28 Days(Baseline up to 6 weeks of double-blind treatment period)
- Clinical Global Impression of Change (CGI-C) Score(Week 6 of double-blind treatment period)
- Change from Baseline in Clinical Global Impression of Severity (CGI-S) Score(Week 6 of double-blind treatment period)
- Number of Participants Reporting Treatment-emergent Adverse Events(Baseline up to 6 weeks of double-blind treatment period)
- Mean Plasma Concentration of CBD(Baseline up to 6 weeks of double-blind treatment period)
- Mean Plasma Concentration of Metabolite 7-OH-CBD(Baseline up to 6 weeks of double-blind treatment period)
- Mean Plasma Concentration of Metabolite 7-COOH-CBD(Baseline up to 6 weeks of double-blind treatment period)