An Open Label, Dose-Escalation Study of Encapsulated Cell Biodelivery of Nerve Growth Factor to the Cholinergic Basal Forebrain of Alzheimer´s Disease Patients
试验速览
- 阶段
- 1 期
- 发起方
- NsGene A/S
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Adverse events
研究概览
简要总结
Cholinergic neurons in the basal forebrain project widely to the cerebral cortex and hippocampus. These neurons depend on nerve growth factor (NGF) from their target areas for survival. Impaired NGF supply is part of the Alzheimer's disease (AD) pathology, and the degeneration of these neurons correlates with the cognitive decline in these patients. The objective of encapsulated cell biodelivery (ECB) is to maintain normal levels of NGF to support cholinergic function. NsGene's NGF secreting ECB device (NsG0202) combines the potential benefits of targeted gene therapy with the safety of a retrievable implantable device.
The study is an open label, single centre, 12-month, dose-escalation phase Ib study in patients with mild to moderate AD. The primary objective is safety and tolerability, while secondary outcomes measure include cognition, behaviour, neuropsychology, activities of daily living (ADL), positron emission tomography (PET) imaging and electroencephalography (EEG).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 50 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 50-80 years
- •All patients must fulfil the NINDNDS-ARDRA criteria of Alzheimer's disease.
- •The subject must have a score between 15-24 inclusive, on the mini-mental state examination (MMSE).
- •The subject must have a caregiver who is able and committed to assist the subject to comply with the trial protocol, and who is willing to provide the information required at assessment interviews.
- •Informed consent must be obtained from the subject together with a close caregiver, in accordance with the requirements of the ethical committee.
排除标准
- •A diagnosis of Schizophrenia, Schizo-affective disorder or paranoid disorder according to DSM IV without any suspicion cognitive decline.
- •Patients with the following co-existing medical conditions:
- •History of seizures.
- •Brain tumor including meningeoma.
- •Clinically significant hepatic, renal, pulmonary, metabolic or endocrine disturbances.
- •Clinically significant back pain.
- •Bleeding disorders.
- •Patients who, in the opinion of the investigator, are otherwise unsuitable for a trial of this type.
研究组 & 干预措施
Nerve Growth Factor
Dose 1
干预措施: Nerve Growth Factor (Drug)
Nerve Growth Factor 2
Dose 2
干预措施: Nerve Growth Factor (Drug)
结局指标
主要结局
Adverse events
时间窗: 12 months
次要结局
- Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-Cog)(12 months)