A Phase 1 Study to Evaluate the Safety and Efficacy of ATHENA CAR-T in Subjects With Moderate or Severe Systemic Lupus Erythematosus
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- EdiGene Inc.
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Frequency of AEs, SAEs, lab abnormalities, AESIs
研究概览
简要总结
The goal of this clinical trial is to test ATHENA CAR-T injection in adults with moderate to severe Systemic Lupus Erythematosus. The main question it aims to answer is:
• To evaluate the safety and tolerability of ATHENA CAR-T.
After screening, participants will be subjected to lymphodepletion regimen. After recovery, participants will be injected with ATHENA CAR-T injection and followed up to 24 months.
详细描述
This study is a single center, one-arm, open label, phase I study aimed at evaluate the safety and effectiveness of ATHENA CAR-T treating moderate to severe SLE patients.
A traditional "3+3" design is used with two doses. DLT is monitored. Safety and effectiveness are followed up until 24 months post infusion of ATHENA CAR-T. Besides safety monitoring, efficacy is evaluated via SLEDAI-2000, BILAG-2004, PGA.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or Female, between 18 and 56 years old;
- •diagnosed with SLE according to 2019 EULAR/ACR SLE classifications;
- •anti-Nuclear Antigen Ab positive (titer NLT 1:80) and/or dsDNA ab positive and/or Anti-Sm ab positive at screening;
- •at screening, SLEDAI-2000 scoring NLT 8 points, if low complement scoring and/or anti-dsDNA ab scoring is available, the SLEDAI-2000 scoring except low complement and anti-dsDNA ab should be NLT 6 points;
- •should be subjected to at least 6 months of standard treatment for SLE, and disease active at least two months before screening;
- •good organ functions;
- •trial participants whose partner is fertile agree to use effective contraceptives til 24 months post transfusion, fertile female participants should have negative urine/blood pregnancy test results (participants who were sterilized or menopause for MT 12 months is not considered fertile);
- •voluntary participates this trial and can comprehend and sign ICF.
排除标准
- •Had or has active malignancy;
- •had been subjected to treatment by CD19 targeted therapy or CAR-T therapy or any gene therapy;
- •within 8 weeks before screening, had CNS disease caused by SLE or non-SLE diseases;
- •within 8 weeks before screening, had lupus crisis;
- •has following kidney diseases: within 8 weeks before randomization, had SLE with serious kidney involvement or need treatment using medications prohibited by protocol to treat active nephritis, or need hemodialysis or need treatment by prednisone MT 100mg/d for longer than 14d or equivalent therapy;
- •had serious allergy to any lymphodepletion medication or ingredients of ATHENA CAR-T;
- •has uncontrolled fungi, bacterial or viral infection or other infections investigator deemed not suitable to participate in the study;
- •combined with other autoimmune disease that needs treatment;
- •pregnant or lactating women;
- •has other factors that deemed not suitable by investigator.
研究组 & 干预措施
ATHENA CAR-T Arm
A conditioning chemotherapy regimen will be administered followed by investigational treatment of ATHENA CAR-T
干预措施: ATHENA CAR-T (Biological)
ATHENA CAR-T Arm
A conditioning chemotherapy regimen will be administered followed by investigational treatment of ATHENA CAR-T
干预措施: Fludarabine (Drug)
ATHENA CAR-T Arm
A conditioning chemotherapy regimen will be administered followed by investigational treatment of ATHENA CAR-T
干预措施: Cyclophosphamide (Drug)
结局指标
主要结局
Frequency of AEs, SAEs, lab abnormalities, AESIs
时间窗: 0 day to 24 months after treatment
Monitor grade and frequency of Adverse Events (AEs), Severe Adverse Events (SAEs), abnormal laboratory findings and Adverse Events of Special Interest (AESI).
Dose Limiting Toxicity
时间窗: 0~28 day after treatment
Dose Limiting Toxicity (DLT) is defined as AEs related to ATHENA CART from infusion till 28 days post infusion.
次要结局
- Efficacy: Percent of patients achieved SRI-4(0 to 16 weeks after treatment)
- Efficacy: Percent of patients' PGA not worsen(0 to 16 weeks after treatment)
- Percent of patients responded by BILAG-2004(0 to 16 weeks after treatment)
- Efficacy: Patients SLEDAI-2000 change compared with baseline(0 to 16 weeks after treatment)
- Efficacy: Patients BILAG-2004 change compared with baseline(0 to 16 weeks after treatment)
- Efficacy: Immunologic parameters(0 day to 24 months after treatment)
- Efficacy: Immunologic parameters (cont)(0 day to 24 months after treatment)