A Multicenter, Open-label, Flexible Dose Study to Assess the Long-term Safety of Pharmaceutical Cannabidiol Oral Solution as an Adjunctive Treatment for Pediatric Subjects With a Treatment-resistant Seizure Disorder Who Complete INS011-14-029 or Part A of INS011-15-054
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 52
- 试验地点
- 11
- 主要终点
- Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values
研究概览
简要总结
This is a multicenter, open-label trial to assess the long-term safety and efficacy of Cannabidiol Oral Solution as adjunctive therapy for pediatric participants with treatment-resistant seizure disorders, including Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS). All participants have rolled over from previous trials: INS011-14-029 (NCT02324673) and INS011-15-054 (NCT02551731).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed activities through Day 11 in INS011-14-029 or Part A (Visit 6) INS011-15-054
- •Informed consent/assent (as applicable) was voluntarily provided by the participant and/or parent(s)/caregiver(s) in accordance with applicable laws, regulations, and local requirements
- •Is medically stable with no anticipated changes in chronic medications in the opinion of the Investigator
- •Continues to meet protocol-specified criteria for qualification and contraception, including treatment-resistant seizure disorder
- •In the opinion of the Investigator, the subject and/or parent(s)/caregiver(s) are able to continue keeping accurate seizure diaries
排除标准
- •Inadequate supervision by parent(s)/caregiver(s)
- •History or current use of dietary supplements, drugs or over-the counter medications outside protocol-specified parameters
- •Signs, symptoms or history of any condition that, per protocol or in the opinion of the investigator, might compromise:
- •the safety or well-being of the participant or study staff
- •the safety or well-being of the participant's offspring (such as through pregnancy or breast-feeding)
- •the analysis of results
研究组 & 干预措施
Infants
Participants 1 to<2 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The maximum daily dose was 40 mg/kg/day.
Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant.
The total daily dose, milligrams per kilograms per day (mg/kg/day), will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion.
干预措施: Cannabidiol Oral Solution (Drug)
Children
Participants 2 to <12 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant.The maximum daily dose was 40 mg/kg/day.
Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant.
The total daily dose (mg/kg/day) will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion.
干预措施: Cannabidiol Oral Solution (Drug)
Adolescents
Participants 12 to <17 years of age. Participants who completed INS011-14-029 initiated this study on the dose with which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant. The maximum daily dose was 40 mg/kg/day.
Participants who enrolled from INS011-15-054 continued treatment with the dose at which they were being treated previously, and dose modifications were made at the Investigator's discretion if tolerability or efficacy issues were observed for a particular participant.
The total daily dose (mg/kg/day) will be evenly split between morning and evening doses (12 hours apart). If tolerability issues arise, the participant's dose may be changed at the investigator's discretion.
干预措施: Cannabidiol Oral Solution (Drug)
结局指标
主要结局
Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Values
时间窗: Up to Week 50
Laboratory values include chemistry and hematology, and urinary analysis.
Percentage of Participants With Adverse Events
时间窗: Up to Week 50
An Adverse Event (AE) is any untoward medical occurrence in a subject administered a pharmaceutical product. It does not necessarily have a causal relationship with this treatment.
Percentage of Participants With Serious Adverse Events
时间窗: Up to Week 50
A serious adverse event is any untoward medical occurrence ( whether considered to be related to investigational product or not) that at any dose results in death, is life threatening, requires inpatient hospitalization, results in disability/incapacity, is a congenital abnormality/ birth defect, or medically significant as determined by an investigator.
Percentage of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG) Findings
时间窗: Up to Week 48
Percentage of Participants With Clinically Significant Change From Baseline in Vital Signs
时间窗: Up to Week 50
Change From Baseline in Trough Plasma Levels of Cannabidiol and Its 7-OH Metabolite
时间窗: Up to Week 50
次要结局
- Vineland Adaptive Behavior Scales (VABS)(Up to Week 48)
- Number of Participants With a Positive Response on the Columbia-Suicide Severity Rating Scale (C-SSRS)(Up to Week 50)