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C-CAR168, an Autologous Anti-CD20/BCMA Chimeric Antigen Receptor T-Cell Therapy, in Participants With Systemic Sclerosis (SSc) Refractory to Standard Therapy

2026年10月7日 更新者:AbelZeta Inc.

A Multi-center, Phase 1b Clinical Study of an Autologous Anti-CD20/BCMA Chimeric Antigen Receptor T-Cell Therapy (C-CAR168) for the Treatment of Systemic Sclerosis (SSc) Refractory to Standard Therapy

This Phase 1b, multicenter, open-label study will evaluate the safety and efficacy of a single infusion of autologous anti-CD20/BCMA chimeric antigen receptor T cells (C-CAR168) following lymphodepleting chemotherapy in participants with refractory Systemic Sclerosis (SSc) who are not responding to standard therapy. Approximately 6-12 participants will undergo leukapheresis, lymphodepletion with fludarabine and cyclophosphamide, and infusion of C-CAR168. Participants will be followed for 24 months (2 years) to evaluate safety, CAR T-cell persistence, pharmacokinetics/pharmacodynamics, biomarkers, and preliminary efficacy results. Long-term safety follow-up for gene therapy-related events will continue for up to 15 years following CAR T-cell infusion.

研究概览

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。

学习联系方式

研究联系人备份

学习地点

    • Arizona
      • Gilbert、Arizona、美国、85234
        • Research Site

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 70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

Key Inclusion Criteria:

  1. Able to sign and date the Informed Consent Form (ICF).

  2. Female or male, aged 18-70 years (inclusive) with body weight ≥40 kg.

  3. Have a clinical diagnosis of diffuse cutaneous SSc according to the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification.

  4. Disease duration between the onset of the first non-Raynaud's sign/symptom and ICF signing shall be ≤ 6 years.

  5. SSc Disease Activity meet all following criteria:

    1. Modified Rodnan Skin Score (mRSS) ≥15.
    2. Chest High Resolution Computed Tomography (HRCT) shows SSc related ground-glass opacities.
    3. Percent predicted forced vital capacity (ppFVC)<70% or precent predicted (diffusing capacity of the lungs for carbon monoxide) ppDLco<70%.
  6. Positivity for at least one SSc-specific autoantibodies:

    1. anti-Scl70 antibody.
    2. anti-RNA polymerase III antibody.
    3. anti-centromere antibody.
    4. anti-Th/To antibody.
    5. and anti-U3RNP antibody.
  7. Refractory to at least two prior standard-of-care regimens, including immunosuppressives and/or a biologic note: "Refractory" is defined as receiving each listed drug for ≥6 months with either no response, disease progression after transient remission, or persistent disease progression on treatment.

  8. Ability to taper steroids to protocol allowed doses

Exclusion Criteria:

  1. ppFVC<45% or ppDLco <40%.

  2. Severe pulmonary arterial hypertension (PAH) as measured by screening echocardiogram (ECHO), or had documented mean positive airway pressure PAP (mPAP) > 45mmHg on recent right heart catheterization (RHC).

    Note: RHC is not mandatory at screening, however participant with high-risk ECHO findings warrant close clinical vigilance, and participant with mPAP> 45mmHg at baseline shall not proceed with lymphodepletion.

  3. Presence of clinically significant abnormalities on chest high-resolution computed tomography (HRCT) that are UNRELATED to SSc.

  4. Cutaneous sclerosis that impedes venous access required for leukapheresis, lymphodepletion, C-CAR168 infusion, other study procedures and emergency rescue interventions.

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:不适用
  • 介入模型:单组作业
  • 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:C-CAR168

Participants will receive: • Leukapheresis • Fludarabine • Cyclophosphamide • Single intravenous infusion of C-CAR168

Autologous anti-CD20/BCMA chimeric antigen receptor T-cell therapy administered as a single intravenous 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
Incidence and severity of treatment-emergent adverse events
大体时间:Through Month 24

Incidence and severity of treatment-emergent adverse events, serious adverse events, dose-limiting toxicities, CRS, ICANS, adverse events of special interest, and treatment- or procedure-related adverse events.

Through Month 24

次要结果测量

结果测量
措施说明
大体时间
Proportion of Participants Achieving Response
大体时间:Through Month 24

Proportion of participants achieving a response per the revised Composite Response Index for Systemic Sclerosis (r-CRISS)

Through Month 24
Pharmacokinetics of C-CAR168 Utilizing Flow Cytometry
大体时间:Through Month 24

Characterize T-cell expansion and persistence utilizing flow cytometry

Through Month 24
Pharmacokinetics of C-CAR168 measured by quantitative polymerase chain reaction (qPCR)
大体时间:Through Month 24

Characterize CAR T-cell expansion and persistence using qPCR

Through Month 24
Proportion of Participants Progressed
大体时间:Through Month 24

Proportion of participants that progress meeting any of the protocol defined progression criteria

Through Month 24
Time to First Response
大体时间:Through Month 24

Time to participant meeting first response as defined in the protocol

Through Month 24
Duration of Response (DOR)
大体时间:Through 24 months

Time of response from first response until progression as defined in the protocol

Through 24 months

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

调查人员

  • 研究主任:Yihong Yao, PhD、AbelZeta 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 (估计的)

2027年3月1日

初级完成 (估计的)

2028年12月1日

研究完成 (估计的)

2030年3月1日

研究注册日期

首次提交

2026年9月27日

首先提交符合 QC 标准的

2026年9月27日

首次发布 (实际的)

2026年10月2日

研究记录更新

最后更新发布 (实际的)

2026年10月13日

上次提交的符合 QC 标准的更新

2026年10月7日

最后验证

2026年10月1日

更多信息

与本研究相关的术语

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)?

不

IPD 计划说明

Individual participant data collected during this study will not be made available to other researchers.

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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