NCT06940336招募中4 期
A Single Arm, Multicenter, Open-label,Phase IV Clinical Study to Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients withTransthyretin Amyloid Polyneuropathy
干预措施
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female participants between the ages of 18 and 80 years.
- •Participant has amyloid deposits confirmed by biopsy (Biopsy must have been performed within 5 years prior to enrollment).
- •Participant must have a TTR mutation that is associated with ATTR-PN (TTR mutation test must have been performed within 5 years prior to enrollment).
- •Participant has peripheral neuropathy at screening.
- •Participant has a Karnofsky Performance Status Score ≥
- •Stages of disease according to symptom severity-stage 1.
排除标准
- •Participant has other causes of amyloidosis, such as light chain amyloidosis, AA amyloidosis.
- •Participant has used tafamidis within 2 months prior to enrollment.
- •Participant has used diflunisal, patisiran, inotersen, or other agents for familial amyloidosis within 30 days prior to enrollment or plan to use them during the study period.
- •Participant has used non-protocol NSAIDs more than 4 times within 30 days prior to enrollment or plan to use them more than 4 times per month during the study period.
- •Participant has used doxycycline,tauroursodeoxycholate within 14 days prior to enrollment or plan to use them during the study period.
- •Participant has sensory motor neuropathy caused by other causes, such as chronic inflammatory demyelinating polyradiculopathy, chronic idiopathic axonal neuropathy, diabetic neuropathy, chronic alcoholic neuropathy, paraneoplastic neuropathy,Guillain-Barre syndrome, vitamin B12 deficiency.
- •Participant has received liver or any other organ except cornea transplantation.
研究组 & 干预措施
Tafamidis Meglumine Soft Capsules
Experimental
干预措施: Tafamidis Meglumine Soft Capsules (Drug)
结局指标
主要结局
Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72
时间窗: Baseline to 72 Weeks
Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72
次要结局
- Change from Baseline NIS-LL at Week 24 and Week 48(Baseline to 24 and 48 Weeks)
- Change from Baseline Modified Body Mass Index(mBMI) at Week 4, Week 8, Week 12,Week 24, Week 36,Week 48 and Week 72(Baseline to 4, 8, 12, 24, 36, 48 and 72 Weeks)
研究者
研究点 (1)
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