NCT07401121招募中1 期
Koanewa: A First in Human, Phase 1b, Open-label, Non-randomised, Single Dose Study to Assess the Safety and Tolerability of CTx1000 in Participants Diagnosed With Amyotrophic Lateral Sclerosis
Celosia Therapeutics Pty Ltd1 个研究点 分布在 1 个国家目标入组 15 人开始时间: 2026年1月12日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants
研究概览
简要总结
This clinical study is in participants with Amyotrophic Lateral Sclerosis and is designed to evaluate the safety and tolerability of the gene therapy CTx1000.
详细描述
CTx1000 is an investigational gene therapy that encodes a degron for targeted degradation of TDP-43 following a single dose intra cisterna magna (ICM) delivery in participants diagnosed with Amyotrophic Lateral Sclerosis (ALS).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 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
- •Diagnosis of ALS in accordance with the revised El Escorial criteria and TRICALS risk score
- •An overall disease duration of ≤ 2 years after the participant's first symptoms
- •No or low circulating anti-AAV9 antibodies (titre ≤ 1:50
- •Stable dosing with a standard of care ALS medication (eg, riluzole and edaravone) and other prescription medications for 30 days prior to Screening
- •Not pregnant or breastfeeding, or willing to cease breastfeeding
- •All participants must use a barrier method of contraception
排除标准
- •Any participants with genetic forms of ALS, including C9ORF72 repeat carriers, except for TARDBP gene variants, as confirmed by previous clinical history genetic testing
- •Any history of myocardial infarction or stroke within 6 months prior to Screening, or uncontrolled diabetes (HbA1C > 9%)
- •Positive test for cytomegalovirus, hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody.
- •Inadequate organ function
- •Any participant with a current open tracheostomy
研究组 & 干预措施
Active drug-CTx1000
Experimental
All participants will receive only one dose of the study drug
干预措施: AAV9 Gene therapy (Drug)
结局指标
主要结局
To evaluate the safety and tolerability of CTx1000 in ALS diagnosed participants
时间窗: 52 weeks
Incidence of Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
次要结局
- To evaluate the long-term safety and tolerability of CTx1000 in ALS diagnosed participants(3 years)
- To evaluate the long-term pharmacodynamics and immunogenicity of CTx1000 in ALS diagnosed participants(3 years)
- To evaluate the long-term efficacy of CTx1000 in ALS diagnosed participants(3 years)
研究者
研究点 (1)
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