An Open Label Individual Patient Study Investigating the Safety and Efficacy of GCAR1 in a Patient With Metastatic Relapsed Alveolar Soft Part Sarcoma
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Treatment response
研究概览
简要总结
A single patient study to determine whether GCAR1 is safe and effective for refractory, progressive metastatic alveolar soft part sarcoma (ASPS).
详细描述
CLIC-YYC-GPNMB-05 is an Open Label Individual Patient (OLIP)/Single Patient Study (SPS) developed according to the Health Canada template and guidelines released in 2019 for studies to access therapies not otherwise available to patients, in the situation where there are no options of treatment or cure remaining. The patient under consideration for CLIC-YYC-GPNMB-05 has refractory, progressive metastatic alveolar soft part sarcoma (ASPS). There are no standard therapies for metastatic ASPS known to provide potential for cure, and there are no clinical trials available in Canada for consideration. The investigators propose to treat the patient with GCAR1, a patient-specific cell therapy product containing a mixture of autologous lymphocytes transduced with a lentiviral vector containing a chimeric antigen receptor (CAR) that enables the specific targeting towards tumor cells expressing the cell surface protein glycoprotein non-metastatic B (GPNMB).
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •The patient must have relapsed ASPS that is in the opinion of the treating physician not resectable, or that resection would be associated with significant morbidity.
- •The patient must provide informed consent.
- •The only other eligibility criteria is adequate organ function, defined as creatinine clearance >30 ml/min and LVEF >45%.
排除标准
- •Any active uncontrolled infection
- •Any anti-cancer therapy within 21 calendar days prior to the first dose of lymphodepleting chemotherapy
研究组 & 干预措施
GCAR1
The patient may receive two separate intravenous infusions of cryopreserved, autologous GCAR1, each containing a total of 5.0E6 CAR+ T cells/kg patient body weight. Both infusions will be preceded by standard lymphodepleting chemotherapy (Fludarabine 40 mg/m2 x 3 days, cyclophosphamide 600 mg/m2 x 2 days). The patient will only be eligible to receive a second dose if they had a partial response (PR), stable, or progressive disease (SD/PD) by RECIST 1.1 criteria after receiving Dose 1. A second infusion should not be administered if the patient achieves and sustains a complete response on disease assessment. Dose 2 will be administered at physician discretion and must be given at minimum 60 days and a maximum of 730 days post administration of Dose 1. For the patient to proceed with a second infusion, any toxicity that does not meet the criteria of a Dose Limiting Toxicity must be assessed for clinical significance, and taken into consideration by the PI before proceeding.
干预措施: GCAR1 (Biological)
结局指标
主要结局
Treatment response
时间窗: Diagnostic imaging (CT and/or MRI) will be performed at baseline (pre-treatment) and then subsequently at day 46 and day 91 after GCAR1 infusion, and at 6 months, 9 months and 12 months after last infusion to evaluate response to therapy.
The overall response assessment considers the response of the target and non-target lesions and development of new lesions.
次要结局
未报告次要终点