An Open-label Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of LUCAR-G79D in Subjects With Relapsed/Refractory Systemic Lupus Erythematosus (r/r SLE) and Idiopathic Inflammatory Myopathies (r/r IIM)
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 38
- 试验地点
- 5
- 主要终点
- Incidence, severity, and type of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
This is a prospective, single-arm, open-label and dose-escalation investigator initialed study to evaluate LUCAR-G79D in adult subjects with r/r SLE and r/r IIM.
详细描述
This is a prospective, single-arm, open-label clinical study to evaluate the safety, tolerability, pharmacokinetics and efficacy profiles of LUCAR-G79D, a chimeric antigen receptor (CAR) -T cell therapy in subjects with r/r SLE and r/r IIM. Patients who meet the eligibility criteria will receive LUCAR-G79D infusion. The study will include the following sequential stages: screening, pre-treatment (lymphodepleting chemotherapy), treatment (LUCAR-G79D infusion) and follow-up.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects voluntary agreement to provide written informed consent.
- •Aged 18 to 70 years, either sex.
- •Adequate organ function meet screening criteria.
- •Positive test for cluster of differentiation antigen 19 (CD19).
- •Have been diagnosed of SLE at least 6 months before screening.
- •At screening, antinuclear antibody, and/or anti-dsDNA antibody, and/or anti-Smith antibody should be positive.
- •Fulfill relapsed/refractory SLE conditions.
- •Have been diagnosed of IIM before screening.
- •Positive test for myositis-associated antibodies or myositis-specific antibodies at screening.
- •Fulfill relapsed/refractory IIM conditions.
排除标准
- •Active infections such as hepatitis and tuberculosis.
- •Other autoimmune diseases.
- •Serious underlying diseases such as tumor, uncontrolled diabetes.
- •Female subjects who were pregnant, breastfeeding.
- •Those with a history of major organ transplantation.
研究组 & 干预措施
Chimeric antigen receptor T cells (LUCAR-G79D)
Each subject will be given a single-dose LUCAR-G79D cells infusion at each dose level.
干预措施: LUCAR-G79D T cells (Biological)
结局指标
主要结局
Incidence, severity, and type of treatment-emergent adverse events (TEAEs)
时间窗: 2 years after LUCAR-G79D infusion (Day 1)
Incidence of dose-limiting toxicity (DLT)
时间窗: 2 years after LUCAR-G79D infusion (Day 1)
Maximum concentration (Cmax) in peripheral blood
时间窗: 2 years after LUCAR-G79D infusion (Day 1)
Time of maximum concentration (Tmax) in peripheral blood
时间窗: 2 years after LUCAR-G79D infusion (Day 1)
Area under the concentration-time curve (AUC) in peripheral blood
时间窗: 2 years after LUCAR-G79D infusion (Day 1)
Recommended Dose regimen finding
时间窗: 2 years after LUCAR-G79D infusion (Day 1)
次要结局
- Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) scores from baseline up to 52 weeks for r/r SLE subjects(52 weeks after LUCAR-G79D infusion (Day 1))
- Change in manual muscle testing (MMT-8) scores from baseline up to 52 weeks for r/r IIM subjects(52 weeks after LUCAR-G79D infusion (Day 1))
- Changes from baseline in peripheral blood immunoglobulins from baseline up to 52 weeks for all subjects(52 weeks after LUCAR-G79D infusion (Day 1))
- Changes from baseline in anti-drug antibody from baseline up to 52 weeks for all subjects(52 weeks after LUCAR-G79D infusion (Day 1))