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评估VTx-002在ALS参与者中的安全性和耐受性的研究 (ALS)

2026年11月5日 更新者：Vector Y Therapeutics

肌萎缩侧索硬化症新型实验方案1/2期研究（Pioneer-ALS）：一项开放标签、非对照、多中心研究，旨在评估两种剂量VTx-002的安全性和耐受性

PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS).

Two escalating dose (low dose and high dose) cohorts are planned. The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant. The screening period may last up to 5 weeks to complete screening procedures.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

All participants will receive a single injection of the study drug. During the first year of the study there will be 12 visits to the study center, including an overnight stay after dosing of at least 1 night. There will be a further 4 remote visits (telephone or video call).

From Year 2-5 there will be 8 further visits. These will be every 6 months and will be either in-person at the study site or remote (telephone or video call) if needed or preferred.

Throughout the 5-year observation period, there will be up to 20 study visits to complete follow-up tests and assessments and monitor the ongoing effects of the study drug.

研究类型

介入性

注册 (估计的)

36

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：Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
  • 电话号码：patients@vectorytx.com
  • 邮箱：patients@vectorytx.com

学习地点

  • 比利时
      • Leuven、比利时
        • 招聘中
        • UZ Leuven
        • 接触：
        • 接触：
        • 首席研究员：
          • Dr Philip Van Damme, Doctor of Medicine
  • 美国
    • Arizona
      • Phoenix、Arizona、美国、85013
        • 尚未招聘
        • St Joseph's Hospital and medical Center - Barrow Neurological Institute
        • 首席研究员：
          • Dr Shafeeq Ladha, Doctor of Medicine
        • 接触：
    • California
      • San Diego、California、美国、92121
        • 招聘中
        • University of California San Diego Medical Center
        • 接触：
        • 首席研究员：
          • Dr John Ravits, Doctor of Medicine
    • Florida
      • Jacksonville、Florida、美国、32224
        • 招聘中
        • Mayo Clinic in Florida
        • 接触：
        • 首席研究员：
          • Dr Bjorn Oskarsson, Doctor of Medicine
    • Massachusetts
      • Boston、Massachusetts、美国、02114
        • 招聘中
        • Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
        • 首席研究员：
          • Dr Doreen Ho, Doctor of Medicine
        • 接触：
    • New York
      • New York、New York、美国、10032
        • 尚未招聘
        • The Eleanor & Lou Gehrig ALS Center
        • 首席研究员：
          • Dr Neil Schneider, Doctor of Medicine
        • 接触：
    • Pennsylvania
      • Philadelphia、Pennsylvania、美国、19104
        • 招聘中
        • University of Pennsylvania
        • 首席研究员：
          • Dr Colin Quinn, Doctor of Medicine
        • 接触：
  • 英国
      • Sheffield、英国、S10 2JF
        • 招聘中
        • Royal Hallamshire Hospital
        • 接触：
  • 荷兰
      • Utrecht、荷兰
        • 招聘中
        • UMC Utrecht
        • 接触：
        • 首席研究员：
          • Prof Leonard Van Den Berg, Doctor 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

18年 及以上 (成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Key Inclusion Criteria:

  1. Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements
  2. Male or female ≥ 18 years of age
  3. Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported; clinically probable, clinically definite)
  4. Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests.
  5. A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness of breath)
  6. Erect (seated) SVC % predicted ≥ 80% at Screening
  7. Treatment Research Initiative to Cure ALS (TRICALS) risk score between -2 and -6 at Screening
  8. Has a reliable caregiver/partner/legal representative willing and able to support the participant in participation in the study and to give informed consent on behalf of the participant in the case that disease progression prevents the participant of giving consent (local legal rules will apply).
  9. Treatment with riluzole and/or edaravone is allowed if treatment was started and has remained at a stable dose for at least 2 weeks (riluzole) or one treatment cycle (edaravone) before the Screening visit
  10. Women of childbearing potential (WOCBP) and male participants with female partners who are WOCBP must agree to use highly effective contraception during and after the study. WOCBP cannot be pregnant or breastfeeding
  11. Women of nonchildbearing potential must be post-menopausal or surgically sterile (e.g. hysterectomy, bilateral tubal ligation, ovaries removed)

Key Exclusion Criteria:

  1. Diagnosis of a significant CNS or peripheral nervous system disease other than ALS that may be a cause for the participant's ALS symptoms or may confound study objectives
  2. Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality
  3. Presence of tracheostomy and feeding tube at Screening
  4. Contraindications to corticosteroid use (e.g. due to osteoporosis, uncontrolled blood pressure, diabetes or cholesterol).

5. Significant concomitant disease or condition within 6 months of Screening that could pose an unacceptable safety risk to the participant or interfere with the participant's ability to comply with study procedures, e.g. heart disease, uncontrolled diabetes, liver disease, autoimmune diseases needing strong immune-suppressing drugs, cancer, etc or a current psychiatric diagnosis.

6. Clinically significant abnormalities in laboratory test results at Screening for example poor liver or kidney function, abnormal clotting or infections such as Hepatitis or HIV

7. Use of blood thinners (e.g., warfarin, heparin, and novel oral anticoagulants) and being unable to safely stop them before certain study procedures.

8. Contraindications to imaging methods MRI, MRA, CT due to claustrophobia and/or intolerance to contrast agents.

9. Contraindications to general anaesthesia (GA) or deep sedation

10 Positive test for illegal drugs (except prescribed medications or permitted medicinal/recreational marijuana if used responsibly)

11. Generally frail or if the Investigator deems participation in the study would not be in the best interest of the participant or is likely to prohibit further participation during the study

Other protocol-defined inclusion/exclusion criteria may apply

-

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：Gene Therapy Group 1: Dose 1 (Low Dose)

Gene Therapy: VTx-002 18 participants will receive dose 1 administered intra cisterna magna. Dosing of the first 3 participants will be staggered at specific timepoints apart and with a safety monitoring committee review of health-related information in between each participant being dosed.

VTx-002 is a single dose therapy

Drug: Preventative (Prophylactic) Medication: Methylprednisolone
遗传的：VTx-002

An investigational gene therapy targeting a specific protein.

药品：Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone

To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.

其他名称：
  • 泼尼松龙
  • 强的松
实验性的：Gene Therapy Group 2: Dose 2 (High Dose)

Gene Therapy: VTx-002 18 participants will receive dose 2 administered intra cisterna magna.

The participant dosing in group 2 will be staggered as it was in group 1. VTx-002 is a single dose therapy. Drug: Preventative (Prophylactic) Medication - Methylprednisolone
遗传的：VTx-002

An investigational gene therapy targeting a specific protein.

药品：Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone

To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.

其他名称：
  • 泼尼松龙
  • 强的松

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
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
大体时间：Over 5 years

Assessed by reviewing the nature, incidence, severity, relatedness, seriousness and outcome of treatment emergent adverse events.
Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
大体时间：Over 5 years

Assessed by reviewing laboratory values
Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
大体时间：Over 5 years

Assessed by reviewing Magnetic Resonance Imaging (MRI) findings.
Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
大体时间：over 5 years

Assessed by review of Treatment Induced Peripheral Neuropathy Assessment Scale (TNAS)

*TNAS a brief participant-reported questionnaire used to measure the severity and progression of peripheral neuropathy. Each item is rated on a scale of 0 to 10, where 0 means no symptom and 10 means the symptom is as bad as it can possibly be.
over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
大体时间：Over 5 years

Assessed by reviewing cellular responses by analyzing blood samples.
Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
大体时间：Over 5 years

Assessed by reviewing scores of the Columbia Suicide Severity Rating Scale (C-SSRS)

*C-SSRS is a widely used, evidence-based suicide risk assessment tool that helps identify whether someone is at risk for suicide and gauge the level of support needed. A low score is associated with a lower risk level in this brief questionnaire.
Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
大体时间：At month 6 and month 12

Assessed by performing 4 formal interim analyses

  1. 6 months after Gene Therapy Group 1 receive the single dose treatment
  2. 6 months after Gene Therapy Group 2 receive the single dose treatment
  3. 12 months after Gene Therapy Group 1 receive the single dose treatment
  4. 12 months after Gene Therapy Group 2 receive the single dose treatment
At month 6 and month 12

次要结果测量

结果测量
措施说明
大体时间
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
大体时间：At month 6 and month 12

Changes certain blood values
At month 6 and month 12
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
大体时间：over 12 months

Time to assisted permanent ventilation or death
over 12 months
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
大体时间：Over 5 years

Immune responses assessed by analyzing blood samples
Over 5 years
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
大体时间：At month 6 and month 12

Change in Slow Vital Capacity
At month 6 and month 12
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
大体时间：At month 6 and month 12

Changes in CSF values
At month 6 and month 12
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
大体时间：At month 6 and month 12 *ALSFRS-R is a scale used to monitor disease progression. The 12 questions. Questions are scored from 0 to 4, with a maximum score of 48 indicating full function and a minimum score of 0 indicating significant impairment.

Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
At month 6 and month 12 *ALSFRS-R is a scale used to monitor disease progression. The 12 questions. Questions are scored from 0 to 4, with a maximum score of 48 indicating full function and a minimum score of 0 indicating significant impairment.

其他结果措施

结果测量
措施说明
大体时间
To assess changes in biomarkers
大体时间：Over 5 years

Analysis of blood samples to assess viral shedding
Over 5 years
To assess changes in muscle strength assessments from baseline.
大体时间：Measured at month 6 and month 12

Change in muscle strength measured by Hand-held Dynamometry
Measured at month 6 and month 12
To assess disease severity and improvement
大体时间：over 12 months

Disease severity and improvement measured using the Clinical Global Impression (CGI) scales for Severity (CGI-S) and Improvement (CGI-I).

The CGI scales each use a seven-point scale where 1 indicates less severe (CGI-S) or most improved (CGI-I) and 7 indicates more severe illness (CGI-S) or worsening of condition (CGI-I)
over 12 months
To assess immunogenicity of VTx002
大体时间：Over 5 years

Assessed by the review of the immunogenicity to the capsid and transgene by analyzing blood samples.
Over 5 years
To assess changes Health Related Quality of Life
大体时间：Measured at month 6 and month 12

Change in Health Related Quality of Life measured using the EQ5D-L questionnaire.

*The EQ5D-L is a self-completed questionnaire assessing five core dimensions (Mobility, Self-care, Usual Activities, Pain/Discomfort, Anxiety/Depression). It is scored by combining patient responses across the five dimensions and then converting to a single utility score whereby 1.0 indicates perfect health and 0 indicates death
Measured at month 6 and month 12
To assess changes Health Related Quality of Life
大体时间：Measured at month 6 and month 12

Change in Health Related Quality of Life measured using the ALSAQ-5 questionnaire.

*The ALSAQ-5 is a brief, 5-item questionnaire used to measure the impact of Amyotrophic Lateral Sclerosis on a patient's quality of life. Five key areas are rated for difficulties and converted to a total score from 0 (worst) to 100 (best)
Measured at month 6 and month 12

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

Vector Y Therapeutics

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)

2025年12月19日

初级完成 (估计的)

2027年10月15日

研究完成 (估计的)

2027年10月15日

研究注册日期

首次提交

2025年11月19日

首先提交符合 QC 标准的

2025年12月11日

首次发布 (实际的)

2025年12月17日

研究记录更新

最后更新发布 (实际的)

2026年11月6日

上次提交的符合 QC 标准的更新

2026年11月5日

最后验证

2026年9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

  • VTx-002-01-001
  • 2025-522697-37-00 (克蒂斯)
  • PIONEER-ALS (其他标识符：VectoryTherapeutics)
  • 2025 (美国 NIH 拨款/合同)

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

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.

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