Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG)
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
- 试验地点
- 23
- 主要终点
- Proportion of patients with MG Composite improvement of ≥5 points.
研究概览
简要总结
This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient must be at least 18 years of age.
- •Patient must have Generalized Myasthenia Gravis at the time of screening.
- •Concomitant immunosuppressive drugs must be deemed necessary by the investigator.
- •Seronegative Patients are included
排除标准
- •Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient.
- •Patient is pregnant or lactating.
研究组 & 干预措施
Phase IIb Randomized Control Trial
Generalized Myasthenia Gravis
干预措施: Descartes-08 (Drug)
Phase 1b Dose-Escalation
Generalized Myasthenia Gravis
干预措施: Descartes-08 (Drug)
Phase IIa Expansion
Generalized Myasthenia Gravis
干预措施: Descartes-08 (Drug)
结局指标
主要结局
Proportion of patients with MG Composite improvement of ≥5 points.
时间窗: Day 0 to Day 85
In Part 3, the primary objective is to compare the effect of Descartes-08 versus placebo, as measured by the change in MGC score from baseline to Week 12. In Part 1, up to three ascending doses of Descartes-08 will be administered to each of 3 to 6 patients; the patients will be staggered at least 21 days apart and will complete a safety review between each dose. In Part 2, patients will receive up to 6 doses in three different schedules depending on the arm they were enrolled into and will be monitored for up to 1 year to assess the safety and tolerability of a repeated dosing schedule.
次要结局
- Comparison on Descartes-08 versus placebo on MG assessments in crossover patients(Day 0 to Day 85)
- Comparison on Descartes-08 versus placebo on QMG and MG ADL(Day 0 to Day 85)
- Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales(Day 0 to Day 168)
- Change in titer of myasthenia specific autoantibody titers(Day 0 to Day 168)