To evaluate the efficacy and safety of regorafenib in patients with refractory primary bone tumors.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 2
- 主要终点
- EFS - (Event-Free Survival) event-free survival - will be measured from randomization to the occurrence of: death, assertion of disease progression or recurrence, finding a secondary neoplasm.
研究概览
简要总结
To evaluate the efficacy and safety of regorafenib in patients with refractory primary bone tumors
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Age> 9 years and ≤ 21 years at the time of inclusion for the study.
- •Ewing's sarcoma or osteosarcoma confirmed by histopathological examination and the tests performed so far.
- •Treatment failure identified no earlier than 30 days prior to study treatment initiation (at least one subsection must be met for the patient to meet this criterion): a. progression on treatment of I or another line or b. relapse.
- •Giving written, informed consent to participate in the study prior to the commencement of the procedures included in the study protocol, including treatment with regorafenib in accordance with the current legal regulations.
- •Life expectancy of at least 12 weeks from signing the informed consent.
- •Possibility of swallowing the tablet.
- •Consent to use effective contraception throughout the period of regorafenib treatment and at least 2 years after its discontinuation in patients in puberty.
排除标准
- •Failure to meet any of the inclusion criteria.
- •Patients with heart defects and / or cardiac arrhythmias requiring permanent treatment with antiarrhythmic drugs.
- •Other acute or chronic medical conditions, behaviors, or abnormal laboratory values that may increase the risk of participating in this clinical trial or taking the study medication, or may affect the interpretation of the study results, or, in the investigator's opinion, may cause that the patient should not be enrolled in the study.
- •Prior treatment with regorafenib.
- •Pregnancy and breastfeeding.
- •Known hypersensitivity to the drug or any of its ingredients.
- •Taking medications that cannot be used while under regorafenib treatment.
- •Persistent toxicity related to previous therapy, preventing drug incorporation.
- •Diagnosis of other neoplastic disease prior to inclusion in the study.
- •Patients with uncontrolled hypertension.
- •Patients with diseases related to the coagulation disorders.
结局指标
主要结局
EFS - (Event-Free Survival) event-free survival - will be measured from randomization to the occurrence of: death, assertion of disease progression or recurrence, finding a secondary neoplasm.
EFS - (Event-Free Survival) event-free survival - will be measured from randomization to the occurrence of: death, assertion of disease progression or recurrence, finding a secondary neoplasm.
Determination of the dose of the test substance in patients between 9 and 18 years at which exposure to the drug similar to that recommended for adults will be achieved.
Determination of the dose of the test substance in patients between 9 and 18 years at which exposure to the drug similar to that recommended for adults will be achieved.
To evaluate the safety of regorafenib by analyzing adverse events (AEs) including adverse events of special importance.
To evaluate the safety of regorafenib by analyzing adverse events (AEs) including adverse events of special importance.
Assessment of the safety of regorafenib through the analysis of recorded vital signs, laboratory test results, echocardiography, and ECG.
Assessment of the safety of regorafenib through the analysis of recorded vital signs, laboratory test results, echocardiography, and ECG.
次要结局
- PFS (Progression-Free Survival) - progression-free survival - will be measured from randomization to finding disease progression in imaging studies.
- OS (Overall Survival) - will be measured from randomization to death due to neoplastic disease.
- ORR (Overall Response Rate) - Percentage of patients who achieved a protocol-defined response to treatment.
- Time to reach the target serum concentration of the test substance.
- Maximum serum concentration at steady state Cmaxs.
- Steady-state trough serum concentration Cminss.
- Casual steady-state serum concentration Css.
- Exposure to Ctau.
- Time to steady-state concentration of the test substance in the serum.
研究者
Klinika Onkologii i Chirurgii Onkologicznej Dzieci i Młodzieży IMID
Scientific
Instytut Matki I Dziecka