Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Number of subjects that underwent an autonomic crisis per month
研究概览
简要总结
This is a pilot open-label study to evaluate the feasibility of conducting a clinical trial using sublingual dexmedetomidine sublingual film to treat hyperadrenergic autonomic crises in patients with Familial Dysautonomia at home. The primary aims are to examine the feasibility of performing a clinical trial using dexmedetomidine at home to terminate autonomic crisis, and refine the interventions and assessments used to evaluate autonomic crisis termination.
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 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically confirmed diagnosis of Familial Dysautonomia.
- •Evidence of autonomic crisis, previous treatment with IV dexmedetomidine without significant side effects, and registered medical data within the year preceding the study in our database.
- •One or more autonomic crises during the last year.
- •Age above 18 years.
- •The patient has a responsible caretaker to communicate with the medical providers.
- •Provision of signed and dated informed consent form from the patient and responsible caregiver.
- •Able to state willingness to comply with all study procedures and availability for the duration of the study
- •For males and females of reproductive potential: use condoms for contraception if sexually active.
排除标准
- •At the consideration of the principal investigator, the caregiver cannot fully understand the protocol or communicate during the crisis with the Center.
- •The patient during the crisis, before taking the medication, has any of the following:
- •a. Oxygen saturation less than 92% on room air or baseline need for oxygen, change from baseline oxygen dependency.
- •b. Respiratory rate >20 breaths per minute.
- •c. Supine blood pressure ≤ 90/60mmHg
- •d. Febrile illness with temperature >100.3 F.
- •e. Serological signs of infection (WBC count >10 g/dL, or CRP >10 mg/L or ESR>20, or above their steady historical baseline levels) in recent (less than one month) studies.
- •The patient is a female and has a positive pregnancy test.
- •MoCA score <25 points.
研究组 & 干预措施
Sublingual dexmedetomidine
Participants will be administered two 60 micrograms sublingual films of IGLMI following start of an autonomic crises. The maximum amount for the study is four oral films of 60 micrograms in 24 hours, two at the beginning of the crises and if needed, two additional within two hours.
干预措施: Dexmedetomidine Sublingual (Drug)
结局指标
主要结局
Number of subjects that underwent an autonomic crisis per month
时间窗: Month 6
Number of subjects screened per month
时间窗: Month 6
Number of subjects enrolled per month
时间窗: Month 6
Number of completed crises treated at home within the protocol
时间窗: Month 6
Average duration of the study from visit 1 to the last visit of the last patient
时间窗: From enrollment to end of treatment (up to 6 months)
次要结局
- Change in number of vomiting episodes(Pre-dose, up to 2 hours post-dose)
- Change in blood pressure(Pre-dose, up to 2 hours post-dose)
- Length of time from autonomic crisis onset to the initiation of the video recording(Up to 2 hours)
- Length of time from autonomic crisis onset to administration of the medication(Up to 2 hours)
- Change in heart rate(Pre-dose, up to 2 hours post-dose)
- Change in number of retching episodes(Pre-dose, up to 2 hours post-dose)
- Length of time from autonomic crisis onset to crisis resolution(Up to 24 hours)
- Length of time it took to complete assessments from start of autonomic crisis(Up to 24 hours)
- Percentage of completion of all rating scales(Month 6)