A Prospective, Multicenter, Randomized, Double-blind, Placebo-controlled, Investigator-initiated Phase 2a Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics/Pharmacodynamics (PK/PD), and Exploratory Efficacy of Multiple Doses of NS101 in Patients With Semantic Variant Primary Progressive Aphasia (svPPA), a Subtype of Frontotemporal Dementia (FTD)
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 15
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)
研究概览
简要总结
The purpose of this clinical trial is to evaluate the safety, tolerability, pharmacokinetics/pharmacodynamics (PK/PD), immunogenicity, and efficacy of multiple intravenous administrations of the investigational drug NS101, compared to placebo, in patients with semantic variant primary progressive aphasia (svPPA), a subtype of frontotemporal dementia (FTD)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 55 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants diagnosed at screening with mild to moderate frontotemporal dementia, semantic variant (svFTD), defined by a CDR® Plus NACC FTLD Sum of Boxes (SOB) score between 4.5 and 15.
- •Participants confirmed to be amyloid-negative based on Amyloid PET scan or CSF results within the past 36 months.
排除标准
- •Participants with other degenerative brain diseases as determined by the investigator
- •Participants with other neurological disorders and uncontrolled acute disease
研究组 & 干预措施
NS101 Treatment Group
Participants will receive a two loading dose of NS101 15 mg/kg IV infusion, followed by biweekly dosing for 6 months during the double-blind phase. Those who complete the double-blind phase will be eligible to enter a 3-month open-label extension to receive active treatment.
干预措施: NS101 IV infusion (Drug)
NS101 Placebo Group
Participants will receive a two loading dose of NS101 15 mg/kg IV infusion, followed by biweekly dosing for 6 months during the double-blind phase. Those who complete the double-blind phase will be eligible to enter a 3-month open-label extension to receive active treatment.
干预措施: Placebo IV Infusion (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)
时间窗: Every Visit for 12month(48weeks)
To evaluate the safety and tolerability of repeated administration of NS101 compared to placebo in patients with semantic variant primary progressive aphasia (svPPA), a subtype of frontotemporal dementia (FTD). \<primary efficacy endpoint\> The incidence, causality, and severity of adverse events (AEs), serious adverse events (SAEs), adverse drug reactions (ADRs), and serious adverse drug reactions (SADRs) by group.
次要结局
- Plasma FAM19A5 Concentration(Every Visit for 12month(48weeks))
- Minimum Plasma Concentration of NS101 (Cmin, Ctrough)(Every Visit for 12month(48weeks))
- Anti-Drug Antibodies (ADA)(Every Visit for 12month(48weeks)
- Neutralizing Antibodies (NAbs)(Every Visit for 12month(48weeks))
研究者
Hee-Jin Kim
Professor, Department of Neurology, College of Medicine, Hanyang university
Hanyang University Seoul Hospital