Neuronavigation-guided Focused Ultrasound-induced Blood-brain Barrier Opening in Alzheimer's Disease Patients and Its Effects on Brain Amyloid and Tau
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Total Number of Individuals With Successful Opening of the BBB
研究概览
简要总结
The primary purpose of this phase 1b study is to further assess the safety and reversibility of focused ultrasound induced blood-brain barrier opening (FUS-BBBO) in participants with Alzheimer´s Disease (AD) using a single-element transducer with neuronavigation guidance. Preliminary results from our phase 1a study demonstrate that our neuronavigation-guided FUS system was capable of safely and transiently open the BBB in participants with AD. The information collected in this new study may be used to design future clinical trials to ultimately provide a viable alternative for treatment of AD in a safe and noninvasive manner.
Our secondary objective includes the assessment of the therapeutic efficacy of FUS-BBBO in reducing amyloid beta and neurofibrillary tangles, the main hallmark pathologies of AD, using PET tracers. Based on our preclinical studies in AD transgenic mouse models, FUS-BBBO alone was able to reduce both the amyloid beta and tau protein load, resulting in improvements in behavioral tasks assessing memory. Therefore, in this new study, the effect of FUS-BBBO on the amyloid beta and tau protein load in patients with AD will be assessed through the use of PET tracers.
详细描述
Regarding the primary purpose of this study, the human module for the neuronavigator was implemented in our phase 1a trial, and initial feasibility and safety in a small cohort of AD patients had been tested, with a fast procedural time that required no anesthesia.
Regarding the secondary objective, both amyloid beta and tau protein load in the brain will be assessed at baseline (before FUS-BBBO treatment), and 3-weeks and 3-months after FUS-BBBO treatment, to assess any short-term or long-term changes, respectively.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Device Feasibility
- 盲法
- None
入排标准
- 年龄范围
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Lumason (Drug)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Amyvid (Drug)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: MK-6240 (Drug)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Dotarem (Drug)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Urine test (Diagnostic Test)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Neuronavigation-guided single-element focused ultrasound transducer (Device)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Magnetic Resonance Imaging (MRI) with or without gadolinium contrast agents (Other)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Positron Emission Tomography (PET) (Radiation)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: Blood draw (Diagnostic Test)
Focused ultrasound treatment
Neuronavigation-guided focused ultrasound treatment in Alzheimer's disease patients using a single-element transducer in conjunction with microbubbles.
干预措施: UR5e (Device)
结局指标
主要结局
Total Number of Individuals With Successful Opening of the BBB
时间窗: 4 days, including the day of treatment
Opening the BBB successfully in all the patients, while obtaining a volume of BBB opened being similar and consistent for all patients
次要结局
- Total Number of Safety Events Related to Opening of BBB(4 days, including the day of treatment)
- Percent Change in Amyloid PET Signal Intensity(3 months (±2 weeks))
- Percent Change in MK-6240 PET Signal Intensity(3 months (±2 weeks))