Prospective Randomized Open, Blinded Endpoint (PROBE) Study of AMDX-2011P as a Retinal Tracer in Subjects With Neurodegenerative Diseases Associated With Amyloidogenic Proteinopathy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Amydis Inc.
- 入组人数
- 13
- 试验地点
- 4
- 主要终点
- AMDX-2011P Adverse Events Profile
研究概览
简要总结
The purpose of this research study is to assess safety and tolerability of a single intravenous (given through a vein) dose of the investigational retinal tracer AMDX-2011P in patients with neurodegenerative diseases (Parkinson's disease and ALS).
详细描述
The primary objective of this study is to evaluate the safety and tolerability of three different doses of AMDX-2011P (25mg, 50mg, or 100mg) given as a single intravenous dose in patients with neurodegenerative diseases (Parkinson's disease and ALS). The first cohort of participants taking part in the study will receive the lowest dose of AMDX-2011P (25mg). If no major side effects occur, the dose will be increased for the next group of participants to 50mg and then to 100mg.
Secondary objective of this study is to characterize the pharmacokinetic (PK) profile of AMDX-2011P and AMDX-2011.
Exploratory evaluations of the biological activity of AMDX-2011P in the retina will be performed by imaging.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Diagnostic
- 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
- •For Subjects with Parkinson's Disease
- •Clinically established Parkinson's disease based on Movement Disorder Society (MDS) Clinical Diagnostic Criteria for Parkinson's disease (Table 8) and a modified Hoehn & Yahr scale of 1-3 (Table 9).
- •No suspected atypical parkinsonian syndromes due to drugs, metabolic disorders, encephalitis, or degenerative diseases.
- •For Subjects with ALS
- •Confirmed diagnosis of ALS with both upper and lower motor neuron involvement.
- •For All Subjects
- •Ability to undergo retinal imaging.
- •Subject or legally authorized representative must provide signed informed consent (or signed assent form) prior to study entry and have the ability and willingness to attend and comply with the necessary study procedures and visits at the study site. For subjects unable to physically sign the informed consent, a guardian or trusted care giver can sign on their behalf in presence of an independent witness.
- •Contraception use by study subjects of childbearing potential (male and female) and female partners of childrearing potential male subjects should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
排除标准
- •Presence of any underlying physical or psychological medical condition that would make it unlikely that the subject will complete the study per protocol.
- •Clinically significant laboratory abnormalities assessed by the investigator.
- •Active malignancy and/or history of malignancy in the past 5 years, with the exception of completely excised non-melanoma skin cancer or low-grade cervical intraepithelial neoplasia.
- •Prolonged QTcF (>450 ms for males and >470 ms for females), cardiac arrhythmia, or any clinically significant abnormality in the resting ECG, as judged by the investigator.
- •Presence of any ocular condition that would significantly hinder the ability to detect and quantify hyper-fluorescent puncta (e.g., eyes with significant hyper-autofluorescence that would mask the ability to detect, quantify, and discern post-injection hyper-fluorescent signal from pre-injection hyper-autofluorescence signal).
- •Use of any new prescription therapies or vaccines within 7 days prior to the study drug administration.
- •Drugs with potential phototoxicity per Package Insert are prohibited within 48 hours or 5 half-lives, whichever is longer, prior to first study drug until End-of-study (EOS) visit, except for those required for treatment of underlying disease.
- •Administration of investigational product in another study within 30 days prior to the first study drug administration, or five half-lives, whichever is longer.
- •Females who are pregnant or breastfeeding.
研究组 & 干预措施
AMDX2011P 25mg
25mg (1ml) single bolus injection intravenous for diagnostic review
干预措施: AMDX2011P (Drug)
AMDX2011P 50mg
AMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review
干预措施: AMDX2011P (Drug)
AMDX2011P 100mg
AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review
干预措施: AMDX2011P (Drug)
AMDX2011P 200mg
AMDX2011P 200mg (6-8ml) single bolus injection intravenous for diagnostic review
干预措施: AMDX2011P (Drug)
AMDX2011P 25mg
25mg (1ml) single bolus injection intravenous for diagnostic review
干预措施: AMDX2011P (Drug)
AMDX2011P 50mg
AMDX2011P 50mg (2ml) single bolus injection intravenous for diagnostic review
干预措施: AMDX2011P (Drug)
AMDX2011P 100mg
AMDX2011P 100mg (4ml) single bolus injection intravenous for diagnostic review
干预措施: AMDX2011P (Drug)
结局指标
主要结局
AMDX-2011P Adverse Events Profile
时间窗: 1 week
Incidence of Treatment Emergent Adverse Events (TEAEs) at for each cohort (dose level).
次要结局
- Concentration of AMDX-2011P(8 hours)
- Pharmacokinetic Analysis of AMDX-2011P(8 hours)