Cannabidivarin (CBDV) vs. Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Aberrant Behavior Checklist-Irritability Subscale (ABC-I)
研究概览
简要总结
This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.
详细描述
This clinical research trial aims to study the feasibility and safety of cannabidivarin (CBDV), in children and young adults with Prader-Willi Syndrome (PWS). CBDV has effects independent of Cannabinoid Receptor Type 1 (CB1) and Cannabinoid Receptor Type 2 (CB2) receptor activation and a good safety profile. This proposal addresses the Foundation for Prader Willi Research's PWS Research Plan: Program 1, Clinical Care Research: seeks to evaluate treatments that aim to reduce behavioral symptoms, such as irritability, in order to improve the quality of life of both the individual with PWS and their families. GW Pharmaceuticals (since acquired by Jazz Pharmaceuticals) will provide the CBDV drug and matching placebo.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 5 Years 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Male or Female outpatients aged 5 to 30 years.
- •Diagnosis of PWS confirmed by genetic testing and patient medical records and history.
- •Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to the study start, and for the duration of the study.
- •Have a physical exam and laboratory results that are within the norms for PWS
- •Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the patient's development and behavior throughout the study. Child Assent will be obtained if the subject is 7 years of age or older and has the mental capacity to understand and sign a written assent form and/or give verbal assent.
- •Score on the Clinical Global Impression Scale Severity (CGI-S) ≥ 4 (moderate severity) at baseline.
- •Score of ≥18 on the Aberrant Behavior Checklist-Irritability (ABC-I) at baseline.
- •Agree not to drive or operate machinery.
排除标准
- •Exposure to any investigational agent in the 30 days prior to randomization.
- •Prior chronic treatment with CBD or CBDV.
- •Positive testing for THC or other drugs of abuse via urine testing at the screening visit or baseline visits upon repeat confirmation testing.
- •History of Drug Abuse Disorder including Cannabis Use Disorder
- •A primary psychiatric diagnosis other than PWS, including bipolar disorder, psychosis, schizophrenia, Post-Traumatic Stress Disorder (PTSD), or Major Depressive Disorder (MDD). These patients will be excluded due to potential confounding results.
- •A medical condition that severely impacts the subject's ability to participate in the study, interferes with the conduct of the study, confounds interpretation of study results or endangers the subject's well-being (including but not limited to hepatic or renal impairment and cardiovascular disease).
- •Known or suspected allergy to CBDV or excipients used in the formulation (i.e. sesame).
- •Clinical indications of renal, pancreatic, or hematologic dysfunction as evidenced by values above upper limits of normal for Blood Urea Nitrogen (BUN)/creatinine, values twice the upper limit of normal for serum lipase and amylase, platelets <80,000 /microliter, white blood cell (WBC)<3.0 103 /microliter or > 2x Upper Limit of Normal (UNL) values of aspartate aminotransferase (AST) or alanine aminotransferase (ALT).
- •ECG abnormality at baseline screening or clinically significant postural drop in systolic blood pressure at screening. If the initial screening ECG shows a Bazett's corrected QT interval (QTcB) of greater than 460 msec, then 2 additional ECGs will be conducted in the same sitting, 5 minutes apart. If not recognized at screening, then a full triplicate repeat showing an average QTcB of 460 msec or less to meet all inclusion/exclusion criteria
- •Female subjects who are pregnant will be excluded from the study. If a female subject is able to become pregnant, she will be given a pregnancy test before entry into the study. Female subjects will be informed not become pregnant while taking CBDV. Female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if they become pregnant during the study.
研究组 & 干预措施
Matched Placebo
Weight-based dosing of 10 mg/kg/day of placebo for 12 weeks
干预措施: Placebo (Drug)
Cannabidivarin (CBDV)
Weight-based dosing of 10 mg/kg/day of CBDV for 12 weeks
干预措施: CBDV Compound (Drug)
结局指标
主要结局
Aberrant Behavior Checklist-Irritability Subscale (ABC-I)
时间窗: from Baseline to Week 12
Change in the ABC-I score. The ABC-I is a well-characterized outcome that is accepted by the FDA for the purpose of labeling, and is one of the best and most validated outcome measures in the developmental disabilities. An inclusion cutoff of 18 or higher on the ABC-I at screening was chosen based on multiple medication trials with irritability as the primary target.
次要结局
- Repetative Behavior Scale- Revised (RBS-R)(from Baseline to Week 12)
- Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)(from Baseline to Week 12)
- Hyperphagia Questionnaire for Clinical Trials (HQ-CT)(from Baseline to Week 12)
- Clinical Global Impression Scale - Improvement (CGI-I)(from Baseline to Week 12)
- Caregiver Strain Questionnaire (CSQ)(from Baseline to Week 12)
- Montefiore Einstein Rigidity Scale-Revised-PWS (MERS-R-PWS)(from Baseline to Week 12)
- Aberrant Behavior Checklist (ABC) subscales in lethargy/social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech(from Baseline to Week 12)
- ActiGraph GT9X-BT activity monitors(from Baseline to Week 12)
研究者
Eric Hollander
Director, Autism and Obsessive Compulsive Spectrum Program, Anxiety and Depression Program
Montefiore Medical Center