NCT06397001已完成1 期
An Open-label Single Center, Single Patient Study of an Experimental Antisense Oligonucleotide (ASO) Treatment in AA Amyloidosis
Nelson Leung, MD1 个研究点 分布在 1 个国家目标入组 1 人开始时间: 2023年11月28日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Adverse Events and/or serious adverse events
研究概览
简要总结
The purpose of this study is to assess the safety and efficacy of a subcutaneous injection of nL-SAA1-01in a patient with AA Amyloidosis.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Biopsy proven AA amyloidosis
- •Measurable disease
- •Ability to travel to the study site and adhere to study-related follow-up examinations and/or procedures and provide access to participant's medical records
排除标准
- •Participant has any condition that in the opinion of the site investigator, would ultimately prevent the completion of study procedures
研究组 & 干预措施
nL-SAA1-01
Experimental
Subject will receive subcutaneous injection of nL-SAA1-01
干预措施: nL-SAA1-01 (Drug)
结局指标
主要结局
Adverse Events and/or serious adverse events
时间窗: 1 year
Number of adverse events and/or serious adverse events experienced
次要结局
- Change in kidney function(Baseline, 1 year)
- Change in quality of life - physical well-being(Baseline, 1 year)
- Change in quality of life - spiritual well-being(Baseline, 1 year)
- Initiation of kidney transplantation(1 year)
- Change in quality of life - emotional well-being(Baseline, 1 year)
- Change in quality of life - intellectual well-being(Baseline, 1 year)
- Change in quality of life - overall well-being(Baseline, 1 year)
- Initiation of dialysis(1 year)
研究者
Nelson Leung, MD
Principal Investigator
Mayo Clinic
研究点 (1)
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