Drug Sensitivity Testing and Mutation Profiling in Childhood Sarcomas
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- To correlate results of drug sensitivity and mutation profiles with clinical outcomes in progression-free survival.
研究概览
简要总结
This study is a prospective, non-randomized observational study. Freshly isolated tumor cells will be tested for chemosensitivity to the standard of care drugs as single agents and in combinations using state-of-the-art viability assay designed for ex-vivo high-throughput drug sensitivity testing (DST). In addition, the genetic profile of the tumor will be obtained from the medical records and correlated with drug response.
详细描述
The excised tumors or a biopsy will be interrogated for sensitivity or resistance to FDA-approved and/or available investigational agents. In addition, normal samples (blood or buccal swab) will be collected for genetic analysis of germline mutations and cancer predisposition markers. The timeframe between the sample acquisition and ex vivo DST results return will be approximately 5-10 working days. All drugs tested in the DST assay will be assigned a hybrid score reflecting the tumor's sensitivity and drug toxicity.
This is an observational study and not a treatment protocol. It will assess how ex vivo drug testing and mutation profiling may predict clinical outcomes (response, survival, or relapse). The treating physician will decide which of the standard treatment options is appropriate independent of the DST results. The results of DST will not be available to the treating physician at the time of decision on the treatment regimen. DST will include all drugs from the standard treatment regimens available for all types of sarcomas
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged 21 years or younger at the time of enrollment on this study of any gender, race, or ethnicity.
- •Subjects with suspected or confirmed diagnosis of all types of sarcomas.
- •Subjects who are scheduled for or have recently had biopsy or tumor excised (solid tumors) or bone marrow aspirate (blood cancers).
- •Subjects are willing to have a blood draw or buccal swab done for the purposes of genetic testing.
- •Subjects or their parents or legal guardians willing to sign informed consent.
- •Subjects aged 7 to 17 willing to sign assent.
排除标准
- •Subjects who do not have malignant tissue available and accessible.
- •The amount of excised malignant tissue is not sufficient for ex vivo drug testing and/or genetic profiling.
- •Patients with other types of tumors and tumors that have a high (>90%) cure rate with safe standard therapy.
结局指标
主要结局
To correlate results of drug sensitivity and mutation profiles with clinical outcomes in progression-free survival.
时间窗: Up to 4 years
Progression-free survival (PFS): a composite end point defined as the censored event time from enrollment to either disease relapse or mortality. This will be evaluated retrospectively at the end of the study. The investigators will use the two-sample long-rank test to assess the hazard ratio of PFS events between the two groups classified as drug sensitive and insensitive by the DST.The study will enroll 15 patients and it is assumed 50% of these subjects will be classified as drug sensitive based on the DST at the threshold value of 10.
To correlate results of drug sensitivity and mutation profiles with clinical outcomes in response to therapy.
时间窗: Up to 4 years
Response to therapy (RTT): an event of achieving "partial response" or "complete response" during the study period, based on the best response of the corresponding enrolled patient.
次要结局
- To assess the predictive value of personalized approach in predicting PFS.(Up to 4 years)
- To assess the predictive value of personalized approach in predicting RTT.(Up to 4 years)
研究者
Diana Azzam, PhD
Assistant Professor
Florida International University