An Open-label, Multi-center, Phase 1/2 Study to Assess Safety, Cellular Kinetics and Exploratory Efficacy of Rapcabtagene Autoleucel in Participants With Difficult-to-treat Rheumatoid Arthritis and Severe, Refractory Sjogren's Disease With Organ Involvement
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 27
- 试验地点
- 12
- 主要终点
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability
研究概览
简要总结
A study to assess safety, cellular kinetics and exploratory efficacy of rapcabtagene autoleucel in rheumatoid arthritis and Sjogren's disease
详细描述
The study is intended to assess safety, cellular kinetics and any early sign for efficacy of rapcabtagene autoleucel in participants with difficult-to-treat rheumatoid arthritis (D2T RA) and severe, refractory Sjogren's disease (srSjD) with organ involvement.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent must be obtained prior to participation in the study
- •Men and women aged ≥ 18 years and ≤ 75 years at Screening For RA only
- •Diagnosis of rheumatoid arthritis
- •Treatment failure of standard of care therapies
- •Active disease For SjD only
- •Diagnosis of Sjogren's disease
- •Active disease
排除标准
- •BMI at Screening of ≤17 or ≥ 40 kg/m2
- •Clinically significant active, opportunistic, chronic or recurrent infection
- •Sexually active males unwilling to use a condom during intercourse from the time enrollment
- •Women of childbearing potential, unless they are using a highly effective method of contraception starting from the time of enrollment
- •Female participants who are pregnant, breastfeeding or intending to conceive during the course of the study
- •Inadequate organ function during screening
- •History of lymphoproliferative disease or any known malignancy or history of malignancy
- •History of bone marrow/hematopoietic stem cell or solid organ transplantation
- •Any psychiatric condition or disability making compliance with treatment or informed consent impossible
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Rapcabtagene autoleucel-rheumatoid arthritis
Single infusion of Rapcabtagene autoleucel in participants with rheumatoid arthritis
干预措施: Rapcabtagene autoleucel (Biological)
Rapcabtagene autoleucel- Sjögren's Disease
Single infusion of Rapcabtagene autoleucel in participants with Sjögren's Disease
干预措施: Rapcabtagene autoleucel (Biological)
结局指标
主要结局
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
时间窗: 24 months
Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
时间窗: 24 months
Safety parameters include vital signs, adverse events, laboratory parameters and ECG evaluation
次要结局
- Pharmacokinetic (PK) parameter: Cmax(24 months)
- PK parameter: Tmax(24 months)
- PK parameter: T1/2(24 months)
- PK parameter: Clast(24 months)
- PK parameter: Tlast(24 months)
- PK parameter: AUC(24 months)
- Pharmacokinetic (PK) parameter: Cmax(24 months)
- PK parameter: Tmax(24 months)
- PK parameter: T1/2(24 months)
- PK parameter: Clast(24 months)
- PK parameter: AUC(24 months)
- PK parameter: Tlast(24 months)