CTIS2023-505851-33-00进行中（未招募）1 期
An Open-label, Single-arm Study to Assess the Safety, Pharmacokinetics, and Efficacy of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Participants with Tuberous Sclerosis Complex (1 Month to < 2 Years of Age), Dravet Syndrome (1 Year to < 2 Years of Age), or Lennox-Gastaut Syndrome (1 Year to < 2 Years of Age) who Experience Inadequately-controlled Seizures - GWEP17005
Gw Research Limited0 个研究点目标入组 27 人开始时间: 2023年8月24日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 27
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 17（—）
- 性别
- All
入选标准
- •Participants with TSC (1 month to < 2 years of age), or DS (1 year to < 2 years of age), or LGS (1 year to < 2 years of age) within the specified age range at the time of initial informed consent, Parent(s)/LAR is/are willing to allow the responsible authorities to be notified of participation in the study, if mandated by local law, Parent(s)/LAR is/are willing to allow the participant’s primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the study if the primary care practitioner/consultant is different from the investigator, Participants with TSC must have a diagnosis per the 2012 International Tuberous Sclerosis Complex Consensus Conference. Participants with LGS or DS must have a diagnosis that is consistent with International League Against Epilepsy (ILAE) guidelines and confirmed by the Epilepsy Study Consortium (ESCI), Participants who have uncontrolled seizures, and who are currently receiving 1 or more ASMs., Parent(s)/LAR is/are willing and able to give informed consent for participation in the study, Parent(s)/LAR is/are willing and able (in the investigator’s opinion) to comply with all study requirements (including accurate electronic patient-reported outcome [ePRO] diary completion), Caregiver completes at least 75% of ePRO and paper seizure diary entries during the 28 days of the baseline period (= 21 days of entries), A suitable VEEG, as available in the medical record, within 1 year of Visit 1. When a historical VEEG is not available, and if clinically indicated and appropriate due to uncertainties or new seizures, a VEEG will be completed and read to confirm diagnosis prior to Visit 3. All VEEGs are to be read at baseline by the investigator and an independent reviewer. • A suitable VEEG meets all the following criteria: i. Multichannel (minimum 8-channel) ii. Prolonged continuous recording up to 24 hours iii. Completed within 1 year of Visit 1 iv. Consistent with the participant’s current seizures (in the investigator’s opinion) v. Can be reviewed by the investigator and an independent reviewer prior to Visit 3 vi. Consistent with a diagnosis of inadequately-controlled seizures, Currently taking = 1 ASMs at a dose that remains stable 2 weeks prior to Visit 3 and during the treatment period. Where required for participant safety, adjustments of concomitant ASMs or addition of new ASM may be permitted following discussion with the medical monitor. • Adrenocorticotropic hormone (ACTH) or high dose corticosteroids for the treatment of IS/ES are counted as ASMs., Has seizures that are not adequately controlled through their current ASMs, defined as = 1 seizure reported on the seizure diary during the screening/baseline period.
排除标准
- •Has clinically significant unstable medical condition other than epilepsy, Has any concurrent cardiovascular conditions that will, in the investigator’s opinion, interfere with the ability to assess their ECGs, Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention such as sesame seed oil, Has significantly impaired hepatic function prior to Visit 3, defined as: • Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × upper limit of normal (ULN) and (total bilirubin [TBL] > 2 × ULN or international normalized ratio [INR] > 1.5). • Serum ALT or AST > 5 × ULN. • Serum ALT or AST > 3 × ULN with the presence of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia (> 5%). • Elevated ALT or AST should be discussed with the medical monitor prior to Visit 3; the medical monitor may allow for a confirmatory re-draw prior to Visit 3., Has received another study intervention within 4 weeks prior to Visit 1 or plans to take another study intervention during the study, Caregiver is currently giving or has given recreational or medicinal cannabis, cannabinoid-based medications (including Sativex) or CBD (including Epidiolex/ Epidyolex [GWP42003-P]) to the participant within the 4 weeks prior to Visit 1 or is unwilling to abstain from doing so for the duration of the study, Mother (if breastfeeding) is currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex) or CBD (including Epidiolex/Epidyolex [GWP42003-P]) within the 4 weeks prior to Visit 1 or is unwilling to abstain from doing so for the duration of the study, Has any other clinically significant disease or disorder which, in the opinion of the investigator, may either put the participant, other participants, or site staff at risk because of participation in the study, may influence the result of the study, or may affect the participant’s ability to take part in the study, Any clinically significant abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the study, Has previously been enrolled into this study, Has plans to travel outside their country of residence during the study, unless the participant has confirmation that the study intervention is permitted in the destination country and all stops along the way, Has had clinically significant symptoms or a clinically significant illness within the 4 weeks prior to Visit 1, other than epilepsy, which in the opinion of the investigator could affect seizure frequency, Has undergone general anesthesia within 4 weeks prior to Visit 1, Has undergone surgery for epilepsy within 6 months prior to Visit 1 or has plans to undergo surgery for epilepsy during the study, Has taken felbamate < 1 year prior to Visit 1, Is < 1 year of age and taking valproic acid, Has tumour growth which, in the opinion of the investigator, could affect participant safety, Has clinically significant abnormal laboratory values, in the investigator’s opinion, at screening/baseline, Has clinically significant abnormalities in the ECG measured at screening/baseline
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