Central Mechanisms and Treatment Response of Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 8
- 试验地点
- 2
- 主要终点
- Efficacy of Lumryz
研究概览
简要总结
Using a comprehensive approach of clinico-behavioral testing and neuroimaging, the researchers will examine the clinical effects of the extended-release formulation of sodium oxybate on voice symptoms in spasmodic dysphonia in an open-label, proof-of-concept, dose-finding study.
详细描述
Spasmodic dysphonia (SD), or laryngeal dystonia, is a chronic, debilitating condition that selectively affects speech production due to involuntary spasms in the laryngeal muscles. SD often extends beyond the impairment of vocal communication, causing significant occupational disability and life-long social isolation. Treatment of SD is limited to injections of botulinum toxin into the vocal cords, however, it is often only partially effective and can have side effects. More than half of the people with SD have some relief from drinking alcohol. The previous studies showed that immediate-release sodium oxybate (an oral drug that acts similarly to alcohol) significantly relieves voice symptoms in patients with alcohol-responsive SD. In this study, we will examine the efficacy and safety of extended-release sodium oxybate formulation (Lumryz) as a longer-acting oral agent for the treatment of patients with alcohol-responsive SD.
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
- 21 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provision of signed and dated informed consent form
- •Stated willingness to comply with all study procedures and lifestyle considerations (see Lifestyle Considerations below) and availability for the duration of the study
- •Males and females
- •Age 21-80 years
- •Documented diagnosis of alcohol-responsive laryngeal dystonia
- •Documented positive response to immediate-release sodium oxybate (Xyrem) in prior studies
- •Willingness to adhere to the study intervention regimen
排除标准
- •The incapability of giving informed consent
- •Pregnancy or breastfeeding until a time when they are no longer pregnant or breastfeeding
- •Grade 2 or higher hepatic or renal dysfunction according to the NCI criteria
- •Moderate to severe congestive heart failure
- •Cognitive impairment (MoCA < 26)
- •Past or present suicidal ideations (according to C-SSRS)
- •Alcoholism or high risk for alcohol use disorder according to the NIAAA definition and DSM-5 criteria
- •Asymptomatic presentation due to botulinum toxin treatment until the time they are fully symptomatic and are at least 3 months after the last injection
- •Increased daytime sleepiness (Epworth Sleepiness Scale (ESS>10))
- •Past or present history of any neurological disorders (except for LD and co-occurring voice tremor), such as stroke, movement disorders, brain tumors, traumatic brain injury with loss of consciousness, ataxias, myopathies, myasthenia gravis, demyelinating diseases, alcoholism, drug dependence.
- •Past or present history of any psychiatric problems, such as schizophrenia, major and/or bipolar depression, or obsessive-compulsive disorder
- •Current use of medication(s) affecting the central nervous system
- •Past or present history of brain and/or laryngeal surgery
- •Presence of certain tattoos, ferromagnetic objects in their bodies (e.g., implanted stimulators, surgical clips, prosthesis, artificial heart valve, etc.) that are not MRI comparable and/or cannot be removed for the purpose of MRI study participation
研究组 & 干预措施
Clinical response to sodium oxybate (Lumryz)
Oral administration of sodium oxybate (1.5g, 2.0g, 2.5g, 3.0g)
干预措施: sodium oxybate (Drug)
结局指标
主要结局
Efficacy of Lumryz
时间窗: Through study completion, an average of 4 days
The primary outcome will be the percent change from baseline in LD symptoms 60 min after each dose of drug intake
次要结局
- Duration of treatment efficacy(Through study completion, an average of 4 days)
研究者
Kristina Simonyan
Professor of Otolaryngology-Head and Neck Surgery
Massachusetts Eye and Ear Infirmary