Descartes-08 for Children, Adolescents and Young Adults With Childhood-onset Systemic Lupus Erythematosus, ANCA-associated Vasculitis, Juvenile Myasthenia Gravis, and Juvenile Dermatomyositis
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 3
- 主要终点
- Maximum tolerated dose in Part 1, type and frequency of treatment related SAE's in Part 2
研究概览
简要总结
Safety, tolerability and efficacy of Descarte-08 in children, adolescents and young adults with childhood-onset systemic lupus erythematosus, ANCA-associated vasculitis, juvenile myasthenia gravis, and juvenile dermatomyositis
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least age 12
- •definitive diagnosis of childhood-onset systemic lupus erythematous, juvenile Myasthenie gravis, juvenile dermatomyositis and AAV
- •Signs and symptoms of moderate disease
- •History of systemic treatment
- •Parent/Guardian/Patient must be able to give written informed consent
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;
- •Abnormal PT/INR or PTT increased > 1.5-fold or patient is on anticoagulation therapy (except in cases of elevated PTT with documented lupus anticoagulant; or in patients who have been on stable doses of anticoagulation therapy for more than 6 months of VTE diagnosis; or in patients on stable doses of anticoagulation therapy for at least 8 weeks of atrial fibrillation diagnosis; these conditions will not be exclusionary unless, in the investigator's opinion, they make participation in the study unsafe);
- •ANC < 1000 cells/microliter ;
- •Hemoglobin < 8.0 g/dL ;
- •Platelets < 50,000/mm3 (NOTE: platelet transfusions are permissible);
- •ALT and/or AST with GGT ≥ 3× upper limit of normal
- •Creatine Clearance less than 30mL/min /1.73 m2;
- •History of primary immunodeficiency, organ, or allogeneic bone marrow transplant;
- •Patients must be seronegative for hepatitis B surface antigen;
- •Patients must be seronegative for hepatitis C antibody. If hepatitis C antibody test is positive, then patients must be tested for the presence of viremia by RT-PCR and must be HCV RNA negative;
- •History of positive HIV or positive HIV at screening;
- •Active tuberculosis or positive QuantiFERON test at screening;
- •Any other laboratory abnormality that, in the opinion of the investigator, may jeopardize the subject's ability to participate in the study;
- •Any active significant cardiac or pulmonary disease not related to the primary indication as determined by principal investigator and medical monitor Note: Patients with asthma and COPD controlled with inhaled medications are allowed;
- •Any arterial or venous thromboembolic events in the past 3 months;
- •History of malignancy that required treatment in the past 3 years except for successfully-treated squamous cell and/or basal cell carcinoma of the skin and/or breast or colon cancer that is surgically removed and did not require adjuvant chemotherapy or radiotherapy;
- •Treatment with any investigational agent within 4 weeks of screening or 5 half-lives of the investigational drug (whichever is longer);
- •Receipt of a live vaccination within 4 weeks prior to baseline (Day 1) or intent to receive live vaccination during the study (Note: mRNA-based vaccines such as those against SARS-CoV-2 are not considered live; likewise, the Janssen Covid-19 vaccine is not live);
- •History of significant recurrent infections or any active infection that may interfere with the patient's participation in the opinion of the investigator;
- •Any known psychiatric illness that may interfere with the patient's participation in the study in the opinion of the investigator.
研究组 & 干预措施
Part 1: Decartes-08 to establish Maximum tolerated dose
Intra-patient dose escalation arm with three dose levels over the course of six infusions of cell product.
干预措施: Descartes-08 (Drug)
Part 2: Decartes-08 infusions once weekly for 6 weeks
Descartes-08 infusions at the maximum tolerated dose level from Part 1.
干预措施: Descartes-08 (Drug)
结局指标
主要结局
Maximum tolerated dose in Part 1, type and frequency of treatment related SAE's in Part 2
时间窗: Days 22 and 50 for Part1 , Days 22, 50 and Months 3,6,9 and 12 for part 2
The primary endpoint for Part-1 is the Maximum Tolerated Dose (MTD), defined as the Dose Level at which no more than 20% of the patients treated have shown Dose-Limiting Toxicity (DLT), i.e. at which 3 patients received all 6 weekly infusions without a DLT by Day 50; or 6 patients received 3 weekly infusions with no more than 1 patient having a DLT by Day 50. The primary endpoint for Part-2 is the type and frequency of treatment-related SAEs. This will be assessed on Days 22, 50 and Months 3,6,9,12
Maximum Dose Tolerated
时间窗: Days 2250 for Part 1, Days 11,50, month 3 for part 2 in addition to months, 4,6,9,12
The primary endpoint for Part-1 is the Maximum Tolerated Dose (MTD), defined as the Dose Level at which no more than 20% of the patients treated have shown Dose-Limiting Toxicity (DLT), i.e. at which 3 patients received all 6 weekly infusions without a DLT by Day 50; or 6 patients received 3 weekly infusions with no more than 1 patient having a DLT by Day 50. The primary endpoint for Part-2 is the type and frequency of treatment-related SAEs.
次要结局
未报告次要终点