A Multicenter, Open-Label Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Patients With Prader-Willi Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution
研究概览
简要总结
The objective of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in participants with Prader-Willi Syndrome.
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
- 8 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed activities up to and including Visit 10 (Study Completion) of INS011-16-
- •Participant and/or parent(s)/caregiver(s) fully comprehend the informed consent form and assent form, understand all study procedures, and can communicate satisfactorily with the investigator and study coordinator, in accordance with applicable laws, regulations, and local requirements.
- •If female, is either not of childbearing potential or practicing medically acceptable methods of birth control.
- •Psychotropic treatment will be permitted if the participant has been on a stable dose during the INS011-16-085 and does not anticipate a dose change during the course of the study.
- •Growth hormone treatment will be permitted if the participant has been on a stable dose during INS01-16-
- •Any other treatment including thyroid hormones should be stable prior to entering the INS011-17-115 study.
- •In the opinion of the investigator, the parent(s)/caregiver(s) is (are) willing and able to comply with the study procedures and visit schedules, including venipuncture, and the visit schedules.
排除标准
- •Participant or parent(s)/caregiver(s) have commitments during the study duration that would interfere with attending all study visits.
- •Experienced an anoxic episode related to study drug requiring resuscitation during the previous study.
- •Uncontrolled Type I and Type II Diabetes.
- •Developed an adverse event thought to be related to CBD in the previous study and the investigator determines that continuing treatment with CBD would not be in the best interest of the partient.
- •History of clinically significant gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine, oncologic, pulmonary, immunologic, psychiatric, or cardiovascular disease, or other condition which would jeopardize safety or impact validity of results (per investigator).
- •Currently taking felbamate.
- •Compromised respiratory function or severe respiratory insufficiency.
- •Pregnant or lactating female.
- •In the opinion of the investigator, the participant is unsuitable in any other way to participate in this study.
研究组 & 干预措施
Cannabidiol Oral Solution: 20-40 mg/kg/day
Participants will receive total daily doses between 20 milligrams per kilograms per day (mg/kg/day), 30 mg/kg/day, and 40 mg/kg/day. The two equivalent doses will be administered twice a day with a standard meal approximately every 12 hours.
干预措施: Cannabidiol Oral Solution (Drug)
结局指标
主要结局
Incidence Of Adverse Events (AEs) And Serious Adverse Events (SAEs) Associated With Cannabidiol Oral Solution
时间窗: Up to Week 52
AE is defined as any untoward medical occurrence in a participant administered with a pharmaceutical product which does not necessarily have a causal relationship with the treatment. An SAE is defined as any event that results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or a congenital anomaly/birth defect. A summary of other non-serious AEs and all serious AEs regardless of causality, is located in the Reported AE section.
次要结局
未报告次要终点