Risperidone for the Treatment of Huntington's Disease Chorea
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Maximal Chorea (UHDRS TMC) Score
研究概览
简要总结
The purpose of this study is to assess the safety and benefit of risperidone for the treatment of chorea (involuntary movements) in Huntington's disease. Risperidone is commonly used in clinical practice to treat chorea, however, it has not been approved by the Food and Drug Administration (FDA) to treat chorea. This study will examine 1) whether the investigators see MRI changes with risperidone treatment and 2) whether sensors applied to the participants body can measure chorea and detect changes in chorea.
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
盲法说明
Outcomes assessor will be masked to the visit.
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Manifest HD (Diagnostic Confidence Level 4 + CAG repeat ≥ 37 or family history of HD)
- •UHDRS Total Maximal Chorea (TMC) ≥ 8
- •UHDRS Total Functional Capacity ≥ 5
- •Subject willing and able to provide written informed consent OR legally authorized representative provides written informed consent and subject provides assent*
- •Between 18 and 65 years of age
排除标准
- •Use of antipsychotic, levodopa, dopamine agonist, monoamine oxidase inhibitor or other disallowed medication in the 30 days prior to the baseline visit (see Section 4.2.5)*
- •Prior non-response to risperidone or intolerability to risperidone (in the investigator's opinion)
- •Allergy or hypersensitivity to risperidone
- •Dysphagia that in the investigator's opinion would preclude participation in the study
- •Active suicidal ideation or psychiatric condition that in the investigator's opinion would preclude study participation
- •QTc > 460 msec for women and QTc > 450 msec for men on 12-lead EKG
- •History of cardiac arrhythmia or congenital long QT syndrome
- •Significant renal impairment (creatinine clearance < 30 mL/min as estimated by the Cockgroft-Gault formula) or hepatic impairment (AST or ALT > 2.5 times upper limit of normal OR alkaline phosphatase or total bilirubin > 2 times upper limit of normal)
- •Active drug or alcohol abuse or dependence
- •Pregnant or breast-feeding
- •Any contraindication to MRI (e.g. pacemakers, aneurysm clips, metallic prostheses, shrapnel fragments, claustrophobia)
- •History of active (clinically significant) skin disorder that would interfere with sensor adherence
- •History of allergic response to adhesives
- •Pacemaker, AICD, or other implantable stimulator
- •Use of an investigational drug in the 30 days prior to the baseline visit
- •Inability to complete study activities, as determined by the study team
- •Clinically significant parkinsonism as determined by expert investigator assessment
研究组 & 干预措施
Risperidone
Participants will initiate risperidone 0.5 mg nightly the day after the baseline visit. Dose assessment will occur at pre-specified intervals during the titration phase (week 2, 3, 4, 6, 7). The investigator will increase the dose by 0.5 mg at the week 2, week 3, week 4, and week 6 visits until either optimal chorea benefit has been obtained, an intolerable adverse event occurs, or the maximum allowable dose (3.0 mg) is reached.
干预措施: Risperidone (Drug)
Risperidone
Participants will initiate risperidone 0.5 mg nightly the day after the baseline visit. Dose assessment will occur at pre-specified intervals during the titration phase (week 2, 3, 4, 6, 7). The investigator will increase the dose by 0.5 mg at the week 2, week 3, week 4, and week 6 visits until either optimal chorea benefit has been obtained, an intolerable adverse event occurs, or the maximum allowable dose (3.0 mg) is reached.
干预措施: BioStamp nPoint device (Device)
结局指标
主要结局
Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Maximal Chorea (UHDRS TMC) Score
时间窗: Baseline to week 12
The UHDRS is a validated assessment of HD. The TMC score is a subset of the overall motor assessment and measures maximal chorea in seven different body regions. The TMC score ranges from 0 to 28 with higher scores indicating more chorea.
次要结局
- Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Total Motor Score(Baseline to week 12)
- Change From Baseline in Mean Epworth Sleepiness Scale (ESS)(Baseline to week 12)
- Change From Baseline in Mean Barnes Akathisia Scale Global Clinical Assessment of Akathisia Item Score(Baseline to week 12)
- Number of Participants With a Change in Clinical Global Impression of Change (CGI)(Baseline to week 12)
- Number of Participants With a Change in Patient Global Impression of Change (PGI)(Baseline to week 12)
- Change From Baseline in Mean Quantitative Motor (Q-Motor) Right Hand Speeded Finger Tapping Variability(Baseline to week 8)
- Change From Baseline in Mean Short Problem Behavior Assessment (Short PBA-S) Irritability and Aggression Score(Baseline to week 12)
- Columbia Suicide Severity Rating Scale( C-SSRS)(Baseline to week 12)
- Change From Baseline in Mean Apathy Scale Score(Baseline to week 12)
- Change From Baseline in Mean Hospital Anxiety and Depression Scale-Depression Score(Baseline to week 12)
- Change From Baseline in Mean Montreal Cognitive Assessment(Baseline to week 12)
- Change From Baseline in Mean Unified Huntington's Disease (HD) Rating Scale Independence Scale Score(Baseline to week 12)
- Change From Baseline in Mean Hospital Anxiety and Depression Scale-Anxiety Score(Baseline to week 12)
- Change From Baseline in MC10-Assessed Mean Gait Cadence(Screening to Week 8)
研究者
Ruth Schneider, MD
Assistant Professor
University of Rochester