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Safety and Efficacy of Intrathecal NTF001 Injection in ALS (NTF-ALS)

2026年5月25日 更新者：Ruijin Hospital

A Study of the Safety and Efficacy of Intrathecal NTF001 Injection in the Treatment of Amyotrophic Lateral Sclerosis

This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intrathecal NTF001 injection, an AAV-mediated human neuron-derived neurotrophic factor gene therapy, in patients with amyotrophic lateral sclerosis (ALS).

12 patients with ALS will be enrolled. Each participant will receive a single intrathecal administration of NTF001 and will be followed for 52 weeks after treatment. The primary outcome measures include treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Secondary outcome measures include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, and neurological function.

This study aims to provide preliminary clinical evidence regarding the safety and potential efficacy of intrathecal NTF001 injection in patients with ALS.

研究概览

地位

主动，不招人

条件

干预/治疗

详细说明

This is a single-arm, open-label, early-phase clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of NTF001 injection in patients with amyotrophic lateral sclerosis (ALS). NTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). The investigational product will be administered once by intrathecal injection.

12 patients with ALS will be enrolled in this study. All eligible participants will receive a single intrathecal administration of NTF001 and will undergo scheduled safety and efficacy assessments during a 52-week follow-up period after treatment.

The primary objective of this study is to assess the safety and tolerability of intrathecal NTF001 injection, primarily by evaluating the occurrence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), clinically significant laboratory abnormalities, vital signs, neurological examinations, and other safety-related findings.

The secondary objective is to explore the preliminary efficacy of NTF001 in patients with ALS. Efficacy assessments will include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), quality-of-life assessments, neurological function, and other exploratory clinical indicators.

This study is expected to provide preliminary clinical evidence regarding the safety, tolerability, and potential therapeutic effects of intrathecal NTF001 injection in patients with ALS, and to support further clinical development of AAV-mediated neurotrophic factor gene therapy for ALS.

研究类型

介入性

注册 (估计的)

12

阶段

  • 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。

学习地点

  • 中国
    • Shanghai Municipality
      • Shanghai、Shanghai Municipality、中国、200025
        • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine

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

Inclusion Criteria:

  • Voluntarily participate in this study and sign the informed consent form.
  • Agree to comply with study procedures and cooperate with all study-related assessments throughout the study.
  • Male or female patients aged 18 to 65 years.
  • Meet the diagnostic and exclusion criteria for amyotrophic lateral sclerosis according to the Chinese Expert Consensus on the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis 2022 issued by the Neurology Branch of the Chinese Medical Association.
  • Have a history of amyotrophic lateral sclerosis of no more than 5 years.

Exclusion Criteria:

  • Mini-Mental State Examination (MMSE) score < 24.
  • Patient Health Questionnaire-9 (PHQ-9) score ≥ 16.
  • Abnormal liver or renal function, defined as AST or ALT > 1.5 × upper limit of normal (ULN), or serum creatinine (Cr) > 1.5 × ULN.
  • Abnormal coagulation function or current use of anticoagulants.
  • Positive infectious disease screening, including positive HBsAg or HBV-DNA, positive HCV-RNA, positive HIV test, or positive syphilis serology.
  • Currently receiving antiviral treatment for hepatitis B or hepatitis C.
  • Unstable or severe systemic diseases, including active tuberculosis, cardiovascular, respiratory, gastrointestinal, urinary, psychiatric or neurological disorders, such as epilepsy, hematological disorders, immune system diseases, or abnormal laboratory findings that, in the opinion of the investigator, make the participant unsuitable for this study.
  • Current or previous history of malignant tumor.
  • History of severe allergic reactions, allergy to contrast agents, or inability to undergo surgical anesthesia.
  • Currently participating in another clinical trial, or participation in another clinical trial within 3 months before screening.
  • Previous receipt of gene therapy before screening.
  • Receipt of stem cell therapy within 6 months before screening.
  • Use of other investigational drugs within 4 weeks before screening or within 5 half-lives of the investigational drug, whichever is longer, or use of any medication that, in the opinion of the investigator, may affect this study.
  • Receipt of a live vaccine within 2 months before screening, or any vaccination within 30 days before screening.
  • History of alcohol dependence or drug addiction, and inability to stop alcohol consumption as instructed during the study.
  • Female participants who are pregnant or breastfeeding.
  • Participants considered unsuitable for enrollment by the investigator.
  • Patients requiring ventilator-assisted ventilation.
  • Patients allergic to the investigational intervention.
  • Patients with obvious signs of dementia.
  • Patients with other psychiatric disorders that may affect disease assessment.
  • Severely obese patients, defined as BMI > 35 kg/m².

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：非随机化
  • 介入模型：顺序分配
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：Low-dose NTF001
Participants in this cohort will receive a single intrathecal administration of low-dose NTF001 （2E+14 vg）injection.
遗传的：NTF001 Injection
NTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). It will be administered once by intrathecal injection to patients with amyotrophic lateral sclerosis (ALS). Participants will receive NTF001 according to the assigned dose cohort and will be followed for safety, tolerability, and preliminary efficacy.
实验性的：High-dose NTF001
Participants in this cohort will receive a single intrathecal administration of high-dose NTF001(4-5 E+14 vg) injection after safety evaluation of the low-dose cohort.
遗传的：NTF001 Injection
NTF001 is an investigational AAV-mediated gene therapy product designed to express human neuron-derived neurotrophic factor (NDNF). It will be administered once by intrathecal injection to patients with amyotrophic lateral sclerosis (ALS). Participants will receive NTF001 according to the assigned dose cohort and will be followed for safety, tolerability, and preliminary efficacy.

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
Incidence of Treatment-Emergent Adverse Events and Serious Adverse Events
大体时间：From administration of NTF001 through Week 52
Safety and tolerability will be assessed by evaluating the incidence, severity, seriousness, and relationship to the investigational product of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Safety assessments will also include clinical laboratory tests, vital signs, physical examinations, neurological examinations, and other clinically significant safety findings.
From administration of NTF001 through Week 52

次要结果测量

结果测量
措施说明
大体时间
Change From Baseline in ALS Functional Rating Scale-Revised Score
大体时间：Baseline through Week 52
The ALS Functional Rating Scale-Revised (ALSFRS-R) will be used to assess functional status in patients with ALS. The total score ranges from 0 to 48, with higher scores indicating better functional status.
Baseline through Week 52
Change From Baseline in ALS Quality of Life Assessment
大体时间：Baseline through Week 52
Quality of life will be assessed using an ALS-specific quality-of-life questionnaire. Changes from baseline will be evaluated during the follow-up period, with higher or lower scores interpreted according to the scoring rules of the selected questionnaire.
Baseline through Week 52
Change From Baseline in ALSAQ-40 Score
大体时间：Baseline through Week 52
The Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) will be used to assess disease-specific quality of life. The questionnaire evaluates multiple domains of daily functioning and well-being in patients with ALS, with higher scores generally indicating worse quality of life.
Baseline through Week 52
Change From Baseline in Norris Scale Score
大体时间：Baseline through Week 52
The Norris Scale will be used to assess neurological function and disease severity in patients with amyotrophic lateral sclerosis. Changes from baseline in the Norris Scale score will be evaluated during the follow-up period, with lower scores indicating greater functional impairment.
Baseline through Week 52

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。

赞助

Ruijin Hospital

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4月1日

初级完成 (估计的)

2028年3月31日

研究完成 (估计的)

2028年3月31日

研究注册日期

首次提交

2026年5月25日

首先提交符合 QC 标准的

2026年5月25日

首次发布 (实际的)

2026年6月1日

研究记录更新

最后更新发布 (实际的)

2026年6月1日

上次提交的符合 QC 标准的更新

2026年5月25日

最后验证

2026年5月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • NTF-ALS

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

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