An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to TARDBP (TDP-43) Genetic Mutation
试验速览
- 阶段
- 1 期
- 状态
- Enrolling By Invitation
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Clinical Functioning
研究概览
简要总结
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.
详细描述
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide (ASO) treatment in participants with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Informed consent/assent provided by the participant (when appropriate), and/or participants parent(s) or legally authorized representative(s)
- •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
- •Genetically confirmed neurological disorder
排除标准
- •Participant has any condition that in the opinion of the Site Investigator, would ultimately prevent the completion of study procedures
- •Use of an investigational medication within less than 5 half-lives of the drug at enrollment
结局指标
主要结局
Clinical Functioning
时间窗: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-006 administration in scores on Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R).
Survival
时间窗: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-006 administration in survival status
Disease Biomarkers
时间窗: Baseline to 12 months
Change from baseline at 12-months post nL-TARDB-006 administration in serum/plasma and CSF neurofilament light chain levels
次要结局
未报告次要终点