C-CAR168, an Autologous Anti-CD20/BCMA Chimeric Antigen Receptor T-Cell Therapy, in Participants With Systemic Sclerosis (SSc) Refractory to Standard Therapy
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
地位
干预/治疗
研究类型
注册 (估计的)
阶段
- 阶段1
联系人和位置
学习联系方式
- 姓名:Kirstin Liechty, Head of Clinical Operations, PhD
- 电话号码:267-566-6081
- 邮箱:kirstin.liechty@abelzeta.com
研究联系人备份
- 姓名:Yihong Liechty, Head of Clinical Operations, PhD
- 电话号码:267-566-6081
- 邮箱:kirstin.liechty@abelzeta.com
学习地点
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Arizona
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Gilbert、Arizona、美国、85234
- Research Site
-
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Key Inclusion Criteria:
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Able to sign and date the Informed Consent Form (ICF).
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Female or male, aged 18-70 years (inclusive) with body weight ≥40 kg.
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Have a clinical diagnosis of diffuse cutaneous SSc according to the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification.
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Disease duration between the onset of the first non-Raynaud's sign/symptom and ICF signing shall be ≤ 6 years.
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SSc Disease Activity meet all following criteria:
- Modified Rodnan Skin Score (mRSS) ≥15.
- Chest High Resolution Computed Tomography (HRCT) shows SSc related ground-glass opacities.
- Percent predicted forced vital capacity (ppFVC)<70% or precent predicted (diffusing capacity of the lungs for carbon monoxide) ppDLco<70%.
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Positivity for at least one SSc-specific autoantibodies:
- anti-Scl70 antibody.
- anti-RNA polymerase III antibody.
- anti-centromere antibody.
- anti-Th/To antibody.
- and anti-U3RNP antibody.
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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.
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Ability to taper steroids to protocol allowed doses
Exclusion Criteria:
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ppFVC<45% or ppDLco <40%.
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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.
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Presence of clinically significant abnormalities on chest high-resolution computed tomography (HRCT) that are UNRELATED to SSc.
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Cutaneous sclerosis that impedes venous access required for leukapheresis, lymphodepletion, C-CAR168 infusion, other study procedures and emergency rescue interventions.
学习计划
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
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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
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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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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
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Characterize T-cell expansion and persistence utilizing flow cytometry |
Through Month 24
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Pharmacokinetics of C-CAR168 measured by quantitative polymerase chain reaction (qPCR)
大体时间:Through Month 24
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Characterize CAR T-cell expansion and persistence using qPCR |
Through Month 24
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Proportion of Participants Progressed
大体时间:Through Month 24
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Proportion of participants that progress meeting any of the protocol defined progression criteria |
Through Month 24
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Time to First Response
大体时间:Through Month 24
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Time to participant meeting first response as defined in the protocol |
Through Month 24
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Duration of Response (DOR)
大体时间:Through 24 months
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Time of response from first response until progression as defined in the protocol |
Through 24 months
|
合作者和调查者
调查人员
- 研究主任:Yihong Yao, PhD、AbelZeta Inc.
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
关键字
其他研究编号
- ABZT-413
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
IPD 计划说明
Individual participant data collected during this study will not be made available to other researchers.
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