A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
- 试验地点
- 14
- 主要终点
- Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)
研究概览
简要总结
This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.
详细描述
This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 4-arm study with an open-label treatment phase in patients with PSP. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open label Treatment Period, and a Follow-up Visit 4 weeks post treatment.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 41 Years 至 86 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of probable progressive supranuclear palsy (PSP)
- •Presence of PSP symptoms for less than 5 years
- •Has a reliable caregiver/informant to accompany the patient to all study visits.
- •Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening
- •Patient must reside outside a skilled nursing facility or dementia care facility at the time of Screening, and admission to such a facility must not be planned. Residence in an assisted living facility is allowed
排除标准
- •Patients must not meet any of the following criteria:
- •Presence of other significant neurological or psychiatric disorders
- •History of clinically significant brain abnormality
- •Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic dysfunction, or moderate to severe resting tremor, responsive to levodopa
- •Known history of serum or plasma progranulin level less than one standard deviation below the normal patient mean
- •Known presence of disease-associated mutation in TARDBP, GRN, CHMPB2, or VCP genes; or any other frontotemporal lobar degeneration causative genes not associated with underlying tau pathology
- •History of clinically significant hematological, endocrine, cardiovascular, renal, hepatic, or gastrointestinal disease
研究组 & 干预措施
TPN-101, Dose A
干预措施: TPN-101, 100 mg/day (Drug)
TPN-101, Dose B
干预措施: TPN-101, 200 mg/day (Drug)
TPN-101, Dose C
干预措施: TPN-101, 400 mg/day (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Assess the safety and tolerability of TPN-101 in patients with progressive supranuclear palsy (PSP)
时间窗: 48 weeks
Incidence and severity of spontaneously reported treatment-emergent adverse events (TEAEs) associated with TPN-101 v. placebo administered for up to 48 weeks in patients with PSP
次要结局
- Assess the pharmacokinetics of TPN-101 as measured by concentrations of TPN-101 in plasma and cerebrospinal fluid (CSF)(48 weeks)
- Assess the pharmacodynamic effect of TPN-101 on neurodegeneration as measured by changes in the levels of CSF and blood neurofilament light (NfL)(48 weeks)
- Assess the clinical effect of TPN-101 as measured by changes in score on the Progressive Supranuclear Palsy Rating Scale (PSPRS)(48 weeks)