Phase 1 Trial of the LSD1 Inhibitor Seclidemstat (SP 2577) With and Without Topotecan and Cyclophosphamide in Patients With Relapsed or Refractory Ewing Sarcoma and Select Sarcomas
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 50
- 试验地点
- 15
- 主要终点
- Safety and tolerability of seclidemstat (SP-2577) as a single agent and in combination with topotecan and cyclophosphamide measured by dose limiting toxicities and adverse events according to CTCAE version 5.0
研究概览
简要总结
Single agent, non-randomized, open label expansion in select sarcoma patients including myxoid liposarcoma and other sarcomas that share similar chromosomal translocations to Ewing sarcoma; AND dose expansion of the combination of seclidemstat with topotecan and cyclophosphamide in patients with Ewing sarcoma
详细描述
The single agent expansion cohort of select sarcoma patients will enroll myxoid liposarcoma patients and patients with other sarcomas that share similar chromosomal translocations to Ewing sarcoma (FET-family translocations), including but not limited to desmoplastic small round cell tumor.
A safety lead-in dose escalation and dose expansion will be conducted assessing the combination of seclidemstat with topotecan and cyclophosphamide in patients with relapsed or refractory Ewing sarcoma.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Myxoid Liposarcoma
Twice-daily administration of oral seclidemstat
干预措施: Seclidemstat (Drug)
Sarcomas with FET-family translocations, including demoplastic small round cell tumors
Twice-daily administration of oral seclidemstat
干预措施: Seclidemstat (Drug)
Ewing sarcoma, combination therapy
Twice daily administration of seclidemstat in combination with cyclophosphamide and topotecan
干预措施: Seclidemstat (Drug)
Ewing sarcoma, combination therapy
Twice daily administration of seclidemstat in combination with cyclophosphamide and topotecan
干预措施: Cyclophosphamide (Drug)
Ewing sarcoma, combination therapy
Twice daily administration of seclidemstat in combination with cyclophosphamide and topotecan
干预措施: Topotecan (Drug)
结局指标
主要结局
Safety and tolerability of seclidemstat (SP-2577) as a single agent and in combination with topotecan and cyclophosphamide measured by dose limiting toxicities and adverse events according to CTCAE version 5.0
时间窗: From screening through at least 30 days after end of treatment, up to approximately 24 months
To evaluate the safety and tolerability of seclidemstat (SP-2577) as a single agent and in combination with topotecan and cyclophosphamide in patients with relapsed or refractory Ewing sarcoma and select sarcomas. Toxicities will be graded in severity per the guidelines outlined in the NCI CTCAE version 5.0. The maximum tolerated dose (MTD) is defined as the dose level immediately below the dose level at which ≥ 2 patients from a cohort of 3 to 6 patients experience a dose-limiting toxicity.
次要结局
- Characterization of the pharmacokinetics of SP-2577 as measured by median half-life(From screening through at least 30 days after end of treatment, up to approximately 24 months)
- Food effects on the pharmacokinetics of SP-2577 as measured in both fasted and fed populations, primarily measured by apparent clearance.(From screening through at least 30 days after end of treatment, up to approximately 24 months)
- Characterization of the pharmacokinetics of SP-2577 as measured by area under the curve(From screening through at least 30 days after end of treatment, up to approximately 24 months)
- Determine the maximum tolerated dose of SP-2577 as determined by dose limiting toxicities measured according to CTCAE version 5.0(From screening through at least 30 days after end of treatment, up to approximately 24 months)
- Characterization of the pharmacokinetics of SP-2577 as measured by peak plasma concentration(From screening through at least 30 days after end of treatment, up to approximately 24 months)
- Characterization of the pharmacokinetics of SP-2577 as measured by apparent clearance of seclidemstat(From screening through at least 30 days after end of treatment, up to approximately 24 months)
- Anti-tumor activity as measured according to RECIST 1.1 criteria based upon radiological assessments.(From screening through at least 30 days after end of treatment, up to approximately 24 months)