A Phase 2, Randomized, Open-label, Controlled Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Comparator in Participants With Severe Refractory Idiopathic Inflammatory Myopathies (IIM)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 21
- 试验地点
- 68
- 主要终点
- Proportion of participants achieving moderate- to-major improvement in Total Improvement Score (TIS) at Week 52
研究概览
简要总结
A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)
详细描述
This is a Phase 2, randomized, active-controlled study. This study comprises two cohorts:
- A lead-in cohort enrolling participants to receive rapcabtagene autoleucel
- A randomized cohort with participants receiving either rapcabtagene autoleucel or a comparator option.
Participants in the comparator arm whose signs and symptoms are not fully controlled may receive rapcabtagene autoleucel treatment once the participant is confirmed to be eligible
After end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None (Outcomes Assessor)
盲法说明
The study investigator and the participant will be unblinded to the study treatment. A blinded assessor will perform the efficacy assessments to minimize bias in data collection.
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
- •Men and women, aged ≥ 18 and ≤75 years, with a diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria
- •Participants who had inadequate response to prior therapy
- •Diagnosed with active disease such as presence of at least 1 of the following criteria: abnormal enzyme levels assessed as secondary to IIM, or EMG demonstrating active disease, DM skin rash, muscle biopsy demonstrating active IIM, or MRI demonstrating active inflammation.
- •Participant must meet criteria for severe myositis such as presence of active muscle weakness.
排除标准
- •Any condition during Screening that could prevent a complete washout of medications or could otherwise make the participant ineligible for anti-CD19 CAR-T therapy and further participation in the study
- •BMI at Screening of ≤17 or ≥40 kg/m2
- •Severe muscle damage at Screening
- •Inadequate organ function
- •Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol
- •Other inflammatory and non-inflammatory myopathies
- •Any medical conditions that are not related to IIM that would jeopardize the ability of the participant to tolerate CD19 CAR-T cell therapy
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Rapcabtagene autoleucel
Single infusion of rapcabtagene autoleucel (YTB323)
干预措施: Rapcabtagene autoleucel (Biological)
Comparator
Investigator choice of treatment as per protocol. (Tacrolimus, Mycophenolate mofetil, Cyclophosphamide, or Rituximab)
干预措施: Active Comparator Option (Other)
结局指标
主要结局
Proportion of participants achieving moderate- to-major improvement in Total Improvement Score (TIS) at Week 52
时间窗: Week 52
The percentage of participants with a TIS of at least 40 at the 52nd week after the start of the study, corresponding to moderate-to-major improvement.
次要结局
- Adjusted annual cumulative glucocorticoid dose up to Week 52(Week 52)
- Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52(Baseline, Week 52)
- Proportion of participants achieving major improvement in TIS at Week 52(Week 52)
- Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52(Baseline, Week 52)