Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Sentence Intelligibility Test (SIT)
研究概览
简要总结
Examine the effects of deutetrabenazine on functional speech and gait impairment
详细描述
This is a two-year, prospective, single-arm study examining the effects of deutetrabenazine on functional speech and gait impairment. Participants will undergo comprehensive evaluations of speech production, fine motor skills, gait, and balance both before and after the initiation of deutetrabenazine. The investigators will utilize a variety of standardized, well-validated assessments as well as 3D motion analysis of both speech and gait parameters to determine the functional impact of deutetrabenazine on speech and motor performance.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of HD with documented CAG repeat ≥ 37
- •UHDRS total maximal chorea score of ≥ 8
- •Able to walk at least 10 meters
- •Medically stable outpatient, based on the investigator's judgment
- •Willing and able to give written informed consent prior to performing any study procedures
- •Have completed at least 10th grade
- •Montreal Cognitive Assessment score ≥ 22 on screening
- •Female subjects of childbearing potential agree to use an acceptable method of contraception from screening through study completion
排除标准
- •Severe depression or suicidal ideation
- •History of suicidal behavior
- •Unstable or serious medical or psychiatric illness
- •Renal or hepatic impairment
- •Severe speech impairment or anarthria
- •Inability to swallow study medication
- •Women who are pregnant or breast feeding
- •History of alcohol or substance abuse within the last 12 months
- •Current use of VMAT2 inhibitor (tetrabenazine, deutetrabenazine, valbenazine)
- •Concurrent participation in any other investigational drug trials
- •EKG QTcF> 500 mse
研究组 & 干预措施
Deutetrabenazine
The mode of administration is oral. Subjects will be started on deutetrabenazine at a dose of 6mg/day. Dosing will be up-titrated in increments of 6mg/day per week to achieve optimal chorea control.
干预措施: Deutetrabenazine (Drug)
结局指标
主要结局
Sentence Intelligibility Test (SIT)
时间窗: Up to 10 weeks
SIT is a measure of speech intelligibility that requires the participant to read aloud 11 sentences that increase in length from 5 to 10 words. Speech intelligibility is indexed in terms of percent intelligible. This is determined by calculating the mean number of correct words transcribed from the SIT transcriptions by three unfamiliar listeners and dividing this by the total number of words in the 11-sentence sample.
Motor Speech Evaluation (MSE)
时间窗: Up to 10 weeks
The MSE will include a set of tasks described by Duffy (2013) that permit consistent rating of perceptual characteristics associated with motor speech disorders. These tasks include vowel prolongation, alternating motion rates, sequential motion rates, and contextual speech (oral reading, picture description, conversational speech). These recordings will be used to complete the perceptual assessment of speech characteristics using the Mayo Clinic classification system.
次要结局
- Functional Joint Kinematics Improvements(Up to 10 weeks)
- Four Square Step Test (FSST)(Up to 10 Weeks)
- Functional Gait Assessment (FGA)(Up to 10 weeks)
- 3-D optical motion capture system recording(Up to 10 weeks)
研究者
Amy E Brown
Assistant Professor
Vanderbilt University Medical Center