A Phase 1/2 Study of Cabiralizumab, an Anti-CSF1 Receptor Antibody, in Patients With Pigmented Villonodular Synovitis (PVNS)/ Diffuse Type Tenosynovial Giant Cell Tumor (Dt-TGCT)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 66
- 试验地点
- 24
- 主要终点
- The Incidence of Grade 3 and Grade 4 Adverse Events (AEs) and Defined as Dose-limiting Toxicities (DLTs) in Phase 1
研究概览
简要总结
This is a phase 1/2 single arm, open-label, safety, tolerability, and PK study of cabiralizumab in PVNS/dt-TGCT patients.
详细描述
A Phase 1/2 study was an open-label, dose escalation and dose expansion study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and preliminary efficacy of cabiralizumab, a CSF1-R monoclonal antibody, inpatients with unresectable diffuse tenosynovial giant cell tumors (TGCT).
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Histologically confirmed diagnosis of inoperable PVNS/ dt-TGCT or potentially resectable tumor that would result in unacceptable functional loss or morbidity as determined by a qualified surgeon or multi-disciplinary tumor board (must be documented in the CRF during screening)
- •Measurable PVNS/dt-TGCT by RECIST 1.1 on MRI
- •ECOG performance status <1
排除标准
- •Prior therapy with an anti-CSF1R antibody
- •Prior therapy with PLX3397 unless discontinued for intolerance (i.e., non-progression on prior kinase inhibitor)
- •Liver function tests (including ALT, AST, and total bilirubin), outside of the range of local laboratory normal at Screening
- •Inadequate organ or bone marrow function
- •History of congestive heart failure or myocardial infarction <1 year prior to first study dose administration
- •Significant abnormalities on ECG at Screening
- •Contraindications to MRI and use of intravenous gadolinium-based contrast agents
- •Creatine Kinase ≥ 1.5x the upper limit of normal
- •Positive test for latent TB at Screening (Quantiferon test)
- •Active known or suspected autoimmune disease
结局指标
主要结局
The Incidence of Grade 3 and Grade 4 Adverse Events (AEs) and Defined as Dose-limiting Toxicities (DLTs) in Phase 1
时间窗: 52 weeks
Number of participants with grade 3 and grade 4 adverse events (AE) defined as dose limiting toxicities (DLTs) in Phase 1
The Incidence of Investigator-assessed, Confirmed Objective Responses (ORR) Per RECIST 1.1 (Phase 2)
时间窗: 52 weeks
Number of confirmed objective responses (ORR) as assessed by the investigator per RECIST 1.1 (Phase 2)
次要结局
- PK Parameters of Cabiralizumab: Area Under Concentration-time Curve (AUC)(52 weeks)
- Maximum Serum Concentration (Cmax).(52 weeks)
- Duration of Response Per RECIST 1.1 in Phase 2(52 weeks)
- Pharmacokinetic Clearance (CL).(52 weeks)
- The Incidence of Clinical Laboratory Abnormalities.(52 weeks)
- Minimum Serum Concentration (Cmin).(52 weeks)
- The Incidence of AEs.(52 weeks)
- The Incidence of ECG Abnormalities.(52 weeks)