An Open-Label, Proof-of-Concept Study to Evaluate the Safety and Treatment Effects of SLS-005 (Trehalose Injection, 90.5 mg/mL for Intravenous Infusion) in Participants With Alzheimer's Disease (AD)
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 3
- 主要终点
- Endpoints for Safety and Tolerability of Treatment
研究概览
简要总结
An open-label, proof-of-concept study to evaluate the safety and treatment effects of SLS-005 in Participants with Alzheimer's Disease (AD) treated once or twice weekly for 52 weeks.
详细描述
This is an open-label proof-of-concept study to evaluate the safety and treatment effects of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) administered by IV infusion in either a once weekly or twice weekly dosing regimen in eligible men and women, ages 50-85 years, who have very mild, mild, or moderate dementia as determined by the Clinical Dementia Rating (CDR) global score and a biomarker-supported diagnosis of AD guided by the National Institute on Aging and Alzheimer's Association (NIA-AA) Research Framework.
The study will enroll approximately 30 eligible participants who will be assigned in a 1:1 ratio to treatment with SLS-005 at a dose of 0.75 g/kg. administered by intravenous (IV) infusion in either a once weekly or twice weekly dosing regimen for up to 52 weeks. Participants will be assigned to 1 of the 2 dosing regimens to achieve approximately equal numbers of participants with mild (CDR 0.5 or 1) and moderate (CDR 2) baseline dementia severity in each dosing regimen. A relatively equal number of participants enrolled in each dosing regimen will be assigned to either the amyloid or tau PET procedure. Tracers to be used will be approved by Sponsor and described in the imaging manual.
Adverse events and results of laboratory and physical evaluations will be collected and assessed throughout the study. Fluid biomarkers associated with AD pathology, brain imaging including amyloid or tau PET and volumetric magnetic resonance imaging (MRI), and measures of cognitive performance, functioning in activities of daily living (ADL), and neuropsychiatric behavioral symptoms will be collected at baseline and at scheduled times throughout the study. Participants who terminate treatment early, will be encouraged to complete all safety and outcome procedures as specified in the protocol's schedule of events.
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 85 Years（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
SLS-005 - Once Weekly
SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion). SLS-005 will be administered as a weight-based dose of 0.75 g/kg by IV infusion once a week over 60 ± 5 minutes for volumes <600 mL or 90 minutes +5 min for volumes >600 mL.
For 52 weeks.
干预措施: SLS-005 - Once Weekly (Drug)
SLS-005 - Twice Weekly
SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion). SLS-005 will be administered as a weight-based dose of 0.75 g/kg by IV infusion twice a week over 60 ± 5 minutes for volumes <600 mL or 90 minutes +5 min for volumes >600 mL.
For 52 weeks.
干预措施: SLS-005 - Twice Weekly (Drug)
结局指标
主要结局
Endpoints for Safety and Tolerability of Treatment
时间窗: 52 weeks
Incidences of treatment-emergent early study and treatment discontinuations.
次要结局
- Endpoints for Treatment Effects on Imaging Biomarkers Associated with AD(26 and 52 weeks)
- Exploratory Endpoints - Treatment Effects in Cognitive Performance(13, 26, 39 and 52 weeks)
- Endpoints for Treatment Effects on CSF Biomarkers Associated with AD(26 and 52 weeks)
- Exploratory Endpoints - Treatment Effects in Activities of Daily Living(13, 26, 39 and 52 weeks)
- Exploratory Endpoints - Treatment Effects in Cognitive Impairment(26 and 52 Weeks.)
- Exploratory Endpoints - Treatment Effects in Behavioral Symptoms and Functioning(13, 26, 39 and 52 weeks)
- Endpoints for Treatment Effects on Volumes of Brain Structures(26 and 52 weeks)
- Exploratory Endpoints - Treatment Effects in Dementia Severity(13, 26, 39 and 52 weeks)