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原发性或继发性进展型多发性硬化症受试者的治疗 (IMPACT-MS)

2026年9月17日 更新者：Tr1X, Inc.

一项评估TRX319在原发性或继发性进展型多发性硬化症受试者中的安全性和初步疗效的1/2a期、开放标签、剂量递增研究

The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary progressive MS.

The main questions the trial aims to answer are the following:

  • Is TRX319 safe when administered to patients with progressive forms of MS?
  • At what dose does TRX319 work the best to treat participants with primary and or secondary progressive MS?
  • Is pre-conditioning (with Bendamustine) needed to allow TRX319 to better treat participants with primary and/or secondary progressive MS?

Participants will be asked to be on study for up 1 year and may receive up to 3 total administrations of TRX319. While on study, participants will have blood tests and other assessments (MRI scans and lumbar punctures) done to understand the safety of TRX319 and how it may benefit their multiple sclerosis.

研究概览

地位

招聘中

条件

干预/治疗

研究类型

介入性

注册 (估计的)

39

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

研究联系人备份

学习地点

  • 美国
    • California
      • La Jolla、California、美国、92037
        • 招聘中
        • UC San Diego
        • 首席研究员：
          • Jennifer Graves, MD
        • 接触：
    • Kansas
      • Kansas City、Kansas、美国、66160
        • 招聘中
        • University of Kansas Medical Center
        • 接触：
    • Minnesota
      • Rochester、Minnesota、美国、55912
        • 招聘中
        • Mayo Clinic
        • 接触：
        • 首席研究员：
          • Michel Toledano, MD
    • Missouri
      • St Louis、Missouri、美国、63110
        • 招聘中
        • Washington University, St. Louis
        • 接触：
    • New York
      • New York、New York、美国、10029
        • 招聘中
        • Mt. Sinai
        • 首席研究员：
          • Fred Lublin, MD
        • 接触：
          • Sarah Myoung, MBS
          • 电话号码：43391 2122413391
    • North Carolina
      • Durham、North Carolina、美国、27710
        • 招聘中
        • Duke University
        • 首席研究员：
          • Suma Shah
        • 接触：
    • Ohio
      • Cincinnati、Ohio、美国、45219
        • 招聘中
        • University of Cincinnati
        • 接触：
    • Washington
      • Seattle、Washington、美国、98122
        • 招聘中
        • Providence Swedish
        • 接触：
        • 首席研究员：
          • Pavle Repovic, MD, PhD

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 至 65年 (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

Inclusion Criteria:

  1. Clinical diagnosis of MS with evidence of PPMS or SPMS according to 2025 McDonald criteria.
  2. Expanded Disability Status Scale (EDSS) range ≥ 2.5 to ≤ 6.5.
  3. Evidence of clinical disability progression within 2 years prior to enrollment.
  4. Documented presence of CSF-restricted OCBs and/or elevated IgG index and/or κ free light chain.
  5. Males and females ≥ 18 and ≤ 65 years of age at time of consent.
  6. Evidence of adequate organ function
  7. Women of child bearing potential have a negative pregnancy test at screening.
  8. Contraceptive use by all participants while on study.
  9. Participants must be able to understand, consent, and be willing and able to complete all specified procedures and visits.
  10. Positive varicella zoster virus titer. Participants who test seronegative for varicella zoster virus IgG antibodies need to complete vaccination ≥ 4 weeks prior to TRX319 infusion.
  11. Participants must be willing to refrain from donating blood for 1 year after TRX319 infusion.

Exclusion Criteria:

  1. MS clinical stability on disease modifying therapy.
  2. Clinical relapse of MS in the 1 year prior to study entry.
  3. Diseases other than MS to explain the first demyelinating event, including aquaporin 4 IgG or myelin oligodendrocyte glycoprotein-IgG seropositivity.
  4. Prior treatment with CAR-T or gene therapy product directed at any target.
  5. Prior treatment with mitoxantrone, cladribine (or other chemotherapies), or alemtuzumab within 2 years prior to TRX319 dose.
  6. Prior treatment with CD20-depleting antibodies within 3 months and prior treatment with Bruton's tyrosine kinase inhibitor (BTKi) and sphingosine 1 phosphate (S1P) modulators within 1 month of TRX319 dose.
  7. Plan to or have received live, attenuated vaccines less than 4 weeks (28 days) prior to TRX319 infusion, and other vaccines less than 2 weeks (14 days) prior to TRX319 infusion.
  8. Serologic status reflecting active hepatitis B or C infection.
  9. Positive serology for human immunodeficiency virus (HIV).
  10. History of progressive multifocal leukoencephalopathy.
  11. Untreated active, or active with documented completed treatment but without a negative chest X-ray that shows no evidence of active tuberculosis, or latent tuberculosis.
  12. Primary immunodeficiency as defined by a known genetic disorder.
  13. History of splenectomy.
  14. Impaired cardiac function or clinically significant cardiac disease.
  15. Previous or concurrent malignancy.
  16. Prior organ transplant, or allogeneic hematopoietic stem cell transplantation or recipient of peripheral blood products < 3 years prior to TRX319 infusion.
  17. Major surgery within 4 weeks prior or planned within 4 weeks after TRX319 administration.
  18. History of any other neurologic disorder or medical condition the Investigator considers would increase the risk for the participant, including seizure disorders.
  19. Life-threatening allergies, hypersensitivity, or documented intolerance to TRX319 drug product excipients.
  20. Subjects that are pregnant, breast feeding or aim to become pregnant during the study period (Subjects must agree to use a highly effective method of contraception).
  21. Serious and/or uncontrolled medical condition that, in the Investigator's judgment, would cause unacceptable safety risk, interfere with study procedures or results, or compromise compliance with the protocol.

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：Cohort 1

Dose Level 1
生物：TRX319

TRX319 is an investigational research cell therapy that may treat and provide long term relief to individuals suffering from Primary Progressive Multiple Sclerosis and/or Secondary Progressive Multiple Sclerosis.
实验性的：Cohort 1A

Dose Level 1 with pre-conditioning
生物：TRX319

TRX319 is an investigational research cell therapy that may treat and provide long term relief to individuals suffering from Primary Progressive Multiple Sclerosis and/or Secondary Progressive Multiple Sclerosis.

药品：Bendamustine

Administration of bendamustine prior to TRX319 infusion
实验性的：Cohort 2

Dose level 2
生物：TRX319

TRX319 is an investigational research cell therapy that may treat and provide long term relief to individuals suffering from Primary Progressive Multiple Sclerosis and/or Secondary Progressive Multiple Sclerosis.
实验性的：Cohort 2A

Dose Level 2 with pre-conditioning
生物：TRX319

TRX319 is an investigational research cell therapy that may treat and provide long term relief to individuals suffering from Primary Progressive Multiple Sclerosis and/or Secondary Progressive Multiple Sclerosis.

药品：Bendamustine

Administration of bendamustine prior to TRX319 infusion
实验性的：Cohort 3

Dose Level 3
生物：TRX319

TRX319 is an investigational research cell therapy that may treat and provide long term relief to individuals suffering from Primary Progressive Multiple Sclerosis and/or Secondary Progressive Multiple Sclerosis.
实验性的：Cohort 3A

Dose level 3 with pre-conditioning
生物：TRX319

TRX319 is an investigational research cell therapy that may treat and provide long term relief to individuals suffering from Primary Progressive Multiple Sclerosis and/or Secondary Progressive Multiple Sclerosis.

药品：Bendamustine

Administration of bendamustine prior to TRX319 infusion

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
To assess the safety and tolerability of TRX319 infusion in subjects with Primary Progressive or Secondary Progressive Multiple Sclerosis.
大体时间：From baseline until 12 months post TRX319 Infusion
  • Number of participants with severity of treatment-emergent adverse events (TEAEs) and treatment-emergent serious adverse events (TESAEs)
  • Rate of Adverse Events of Special Interest (AESIs) in participants
  • The safety of TRX319 determined by negative Replication Competent Lentivirus (RCL) at approximately 3-months, 6-months, and 12-months
From baseline until 12 months post TRX319 Infusion

次要结果测量

结果测量
措施说明
大体时间
To characterize target engagement via reduction of Oligoclonal bands (OCB) and/or normalization of cerebral spinal fluid (CSF) Immunoglobulin G (IgG) index
大体时间：From baseline until 12 months post TRX319 Infusion
  • Reduction of OCB in CSF
  • Reduction and/or normalization of CSF IgG index
From baseline until 12 months post TRX319 Infusion
To evaluate the effects of TRX319 on disease progression/reactivation by gadolinium-enhancing MRI
大体时间：From baseline until 12 months post TRX319 Infusion

No increase in gadolinium-enhanced T1-weighted lesions and no new or enlarging T1- or T2-weighted lesions in brain and cervical spinal cord
From baseline until 12 months post TRX319 Infusion
To evaluate disease response, as measured by the Neurostatus Expanded Disability Status Scale (EDSS)
大体时间：From baseline to approximately 12 months post TRX319 infusion

Measure of disease response based on change in disability and walking score at Weeks 12, 24, 36, and 48.
From baseline to approximately 12 months post TRX319 infusion

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

Tr1X, Inc.

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3月1日

初级完成 (估计的)

2028年8月1日

研究完成 (估计的)

2029年1月1日

研究注册日期

首次提交

2026年3月9日

首先提交符合 QC 标准的

2026年3月12日

首次发布 (实际的)

2026年3月17日

研究记录更新

最后更新发布 (实际的)

2026年9月21日

上次提交的符合 QC 标准的更新

2026年9月17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

  • TRX319-01

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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