FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS (FUNCtion ALS)
2026年9月8日 更新者：Trace Neuroscience, Inc.
A Randomized, Double-Blind, Placebo-Controlled, Phase 1 / 2 Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of TRCN-1023 Administered by Intrathecal Injections to Adult People Living With Amyotrophic Lateral Sclerosis
The FUNCtion Amyotrophic Lateral Sclerosis (ALS) trial is a randomized, double-blind, placebo-controlled Phase 1/2 trial to evaluate the safety and tolerability of TRCN-1023 in adults living with ALS.
TRCN-1023 is an investigational medicine given as a single injection into the fluid surrounding the spine (intrathecal injection).
The trial will also assess how the body processes the drug and whether it shows early signs of benefit over 24 weeks.
研究概览
详细说明
TRCN-1023 is an investigational medicine designed to restore the function of a protein called UNC13A, which becomes disrupted in most people with Amyotrophic Lateral Sclerosis (ALS) due to a breakdown in how nerve cells process genetic information.
This trial is designed to evaluate the safety and tolerability of TRCN-1023 given as a single injection into the fluid surrounding the spine, and to identify the best dose to carry forward into future studies.
TRCN-1023 stays in the brain and spinal cord long enough that it may only need to be administered once every 24 weeks.
The trial will also assess how the body processes the drug, whether it engages its intended target, and its effects on disease progression, physical function, and quality of life.
研究类型
介入性
注册 (估计的)
30
阶段
- 阶段2
- 阶段1
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Trace Neuroscience Clinical Trials
- 电话号码：+1 415-390-9509
- 邮箱：Clinicaltrials@traceneuro.com
学习地点
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Munich、德国
- 招聘中
- TUM Klinikum rechts der Isar
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接触：
- Paul Lingor, MD
- 电话号码：+49 89 4140 4606
- 邮箱：paul.lingor@mri.tum.de
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首席研究员：
- Paul Lingor, MD
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Rostock、德国、18147
- 招聘中
- University Medical Center Rostock
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接触：
- Sophie Fischer
- 电话号码：+49 381 494 149542
- 邮箱：Sophie.fischer@med.uni-rostock.de
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Sheffield、英国、S10 2HQ
- 招聘中
- University of Sheffield
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接触：
- Prof Jonathan Cooper-Knock
- 邮箱：j.cooper-knock@sheffield.ac.uk
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首席研究员：
- Prof Jonathan Cooper-Knock
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Utrecht、荷兰、3508 GA
- 招聘中
- University Medical Center Utrecht
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接触：
- Henk-Jan Westeneng
- 电话号码：+31 (0)88 75 53204
- 邮箱：trialals@umcutrecht.nl
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首席研究员：
- Henk-Jan Westeneng
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
不
描述
Key Inclusion Criteria:
- Adults aged 18 to 75 years
- Diagnosis of ALS (clinically definite, clinically probable, or clinically probable laboratory supported)
- ALS symptoms began within the past 24 months
- Able to perform breathing tests: slow vital capacity (SVC) with consistent results, with breathing capacity of at least 60% of the expected value
- Able and willing to meet all study requirements, including travel to the study site, brain magnetic resonance imaging (MRI) scans, lumbar punctures, and blood draws
- Able and willing to use wearable sensors and complete speech assessments at home
- On a stable dose of approved ALS medication for at least 4 weeks prior to screening
- Capable of providing informed consent
Key Exclusion Criteria:
- Carries a confirmed SOD1 or FUS gene mutation
- Has a tracheostomy or requires continuous assisted ventilation more than 22 hours per day during the preceding 3 months before the first Screening Visit
- Has a contraindication to brain MRI (e.g., pacemaker, metal implants)
- Has a contraindication to lumbar puncture or spinal injection (e.g., blood clotting disorders, certain blood thinners, signs of increased pressure in the brain)
- Has significant abnormal liver, kidney, or blood test results
- Is currently enrolled in another clinical trial or has received an investigational treatment within the past 4 weeks
- Has previously received gene therapy, stem cell therapy, or another Antisense oligonucleotide (ASO) treatment
- Has a clinically significant condition other than ALS that could interfere with study participation
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：四人间
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的：TRCN-1023
Antisense oligonucleotide (ASO) administered by intrathecal injection
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TRCN-1023 administered as an intrathecal injection.
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安慰剂比较：Placebo
Matching Placebo Comparator
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Intrathecal injection of artificial cerebrospinal fluid (aCSF).
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Frequency of Adverse Events
大体时间：24 Weeks
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Number and frequency of adverse events assessed over 24 weeks following a single intrathecal injection of TRCN-1023 or placebo
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24 Weeks
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Plasma Concentration of TRCN-1023
大体时间：Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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Plasma TRCN-1023 concentrations
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Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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Cerebrospinal Fluid (CSF) Concentration of TRCN-1023
大体时间：Day 1 (pre-dose and 24 hours post-dose), Weeks 4, 16, and 24 (up to 4 lumbar punctures post-dose)
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Cerebrospinal fluid (CSF) TRCN-1023 concentrations
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Day 1 (pre-dose and 24 hours post-dose), Weeks 4, 16, and 24 (up to 4 lumbar punctures post-dose)
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Time to Peak Plasma Concentration (Tmax)
大体时间：Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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Time of maximum observed plasma concentration (Tmax) of TRCN-1023
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Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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Peak Plasma Concentration (Cmax) of TRCN-1023
大体时间：Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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Maximum observed plasma concentration (Cmax) of TRCN-1023
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Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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Area Under the Plasma Concentration-Time Curve to Last Measurable Concentration (AUClast) of TRCN-1023
大体时间：Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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Area under the plasma concentration-time curve from time zero to last measurable concentration (AUClast) of TRCN-1023
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Day 1 (pre-dose, 1, 2, 6, and 24 hours post-dose), Weeks 4, 10, 16, and 24
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2026年8月20日
初级完成 (估计的)
2027年9月1日
研究完成 (估计的)
2027年9月1日
研究注册日期
首次提交
2026年6月21日
首先提交符合 QC 标准的
2026年6月24日
首次发布 (实际的)
2026年6月29日
研究记录更新
最后更新发布 (实际的)
2026年9月9日
上次提交的符合 QC 标准的更新
2026年9月8日
最后验证
2026年6月1日
更多信息
与本研究相关的术语
其他研究编号
- TRCN-1023-ALS-101
- 2025 (美国 NIH 拨款/合同：Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
- 2025-523111-11 (EudraCT编号)
- 174619 (其他标识符：Food And Drug Administration (FDA))
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
未定
药物和器械信息、研究文件
研究美国 FDA 监管的药品
是的
研究美国 FDA 监管的设备产品
不
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.