An observational, Prospective, Multicenter Study to evaluate safety of cannabidiol 100 mg/ml oral solution as add-on therapy in patients with for Seizures Associated with Lennox-Gastaut Syndrome (LGS) or Dravet Syndrome (DS)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 50
- 试验地点
- 3
- 主要终点
- To evaluate via the adverse events (AE) profile
研究概览
简要总结
This is an observational, prospective, multicenter study designed to evaluate the safety of cannabidiol 100 mg/ml oral solution as an add-on therapy for patients with seizures associated with Lennox-Gastaut Syndrome (LGS) or Dravet Syndrome (DS). Patients with LGS or DS who meet the eligibility criteria will be screened at Day -7 for inclusion in the study. Written informed consent or assent will be obtained from all patients prior to any study-related procedures. At screening, demographic data, comprehensive medical history (including details of seizures since diagnosis and all antiepileptic drugs [AEDs] used), and vital signs will be collected. Laboratory assessments, including hematology, biochemistry, urinalysis, and urine pregnancy tests (UPT), will be performed, and an ECG will be conducted. Eligible patients who meet all inclusion criteria and none of the exclusion criteria will be assigned a unique patient number. Following a 7-day baseline observation period, investigators will assess the patient’s daily seizure frequency. Patients who continue to meet the eligibility criteria will be randomized and will then begin receiving the investigational product (IP). Prior to receiving the IP on Day 1, patients will complete various questionnaires (SGIC-SD or CGIC-SD, CBCL or ABCL, SCQ, QOLCE or QOLIE-31-P, CGIC or SGIC, PGIC). The cannabidiol oral solution will be taken by patients for a period of 112 days. Clinic visits will occur on Days 28, 56, 84, 112, and Day 123, with a follow-up telephone visit on Day 151. During these visits, assessments will include concomitant medications, epilepsy-related hospitalizations, physical examinations, vital signs, ECG, and laboratory tests (hematology, biochemistry, urinalysis, UPT). Additionally, the same questionnaires will be administered at various points in the study.Any adverse events (AEs) observed during the study, regardless of whether they are attributed to the investigational product, will be documented in the Case Report Form (CRF). All AEs, whether identified by the investigator or reported by the patient, will be recorded, and further patient questioning will be conducted as necessary to gather additional information.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 盲法
- None
入排标准
- 年龄范围
- 2.00 Year(s) 至 65.00 Year(s)（—）
- 性别
- All
入选标准
- •Subject aged two to 65 years, Diagnosed with seizers LGS or DS.
- •Established diagnosis of epilepsy, characterized by focal or generalized seizures.
- •All participants will have active epilepsy that requires treatment with anticonvulsant medication.
- •No episodes of seizure clusters of status epilepticus within 30 days prior to entry into the study.
- •Established symptoms of anxiety with functional impairment.
- •Ability to administer medicine orally.
- •Previous subjects who failed at any point to meet continuation criteria and withdraw early may be considered for re-enrolment by the PI on a case by case basis.
- •Participant or legal caregiver capable of providing informed consent and fully capable of monitoring the subjects disease process and compliance with treatment.
- •Participants who are sexually active, must agree to sexual abstinence, or, to use an approved birth control method for the full duration of study participation.
- •No active use of CBD products within the 14 days prior to screening visit and commitment to only use study product for duration of the study.
- •Must meet laboratory ranges.
- •Females of child bearing potential(FCBP) must have a negative pregnancy test at screening and baseline.
- •while on investigational product (IP) and for at least 28 days after taking the last dose of investigational product.
排除标准
- •Baseline lab tests for liver specific transaminase, ALT, over the upper limit of normal (ULN).
- •Clinically significant ECG abnormalities.
- •Previous allergic or hypersensitivity reactions to cannabidiol.
- •No access to a phone or internet to complete remote visits (in person visits acceptable for participants without devices).
- •Active substance abuse or dependence.
- •Presence of psychotic illness or imminent risk of harm to self or others.
- •Current standing use of benzodiazepines (except as "rescue" medicine).
- •Serious unstable medical or neurologic conditions such as HIV, liver or kidney disease, cancer or diabetes.
- •Presence of Epilepsy Syndrome such as Sturge-Weber Syndrome that will be more suitable as a candidate in alternate research studies.
- •Participation in a previous experimental drug study within 30 days of baseline visit.
- •Cognitive functional capacity or English literacy that is insufficient to assure validity of clinical rating scales.
- •Insufficient capacity of caregiver or legal guardian to understand and appropriately consent for study procedures.
- •No exclusions for existing AEDs will be absolute, though consideration of additional monitoring will be in place for patients taking clobazam or valproate.
- •Pregnant, planning to become pregnant, breast feeding, or failing to use an appropriate method of contraception.
结局指标
主要结局
To evaluate via the adverse events (AE) profile
时间窗: Day-(-7), Day-1,Day-28, Day-56, Day-84, Day-112, Day-123, Day-151
the long term safety and tolerability of Cannabidiol 100 mg/ml as
时间窗: Day-(-7), Day-1,Day-28, Day-56, Day-84, Day-112, Day-123, Day-151
add-on therapy in children and adults with for Seizures Associated
时间窗: Day-(-7), Day-1,Day-28, Day-56, Day-84, Day-112, Day-123, Day-151
With Lennox-Gastaut Syndrome (LGS) or Dravet Syndrome (DS)
时间窗: Day-(-7), Day-1,Day-28, Day-56, Day-84, Day-112, Day-123, Day-151
次要结局
- 1) Percentage change from baseline in total seizure frequency, percentage of participants achieving 25 percent reduction in seizure frequency , 50 percent reduction in seizure frequency, 75 percent reduction in seizers frequency from baseline.(2) Average number of seizure free days in the last 28 days, longest duration of seizure free days in last 28 days, number of events of status epilepticus.)
研究者
Dr Sandeep Bhagawan Patil
Noble hospital and research centre