To evaluate the efficacy and safety of naxitamab in patients with refractory Ewing's sarcoma.
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 24
- 试验地点
- 2
- 主要终点
- This primary end point has been redacted following transparency rules and protection of confidential information.
研究概览
简要总结
Safety assessment of the addition of naxitamab to standard 3-week chemotherapy (CHT) in patients with refractory Ewing's sarcoma (ES)
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Histologically proven Ewing sarcoma of the bone or soft tissues.
- •Consent to the use of effective contraception throughout the period of the study and a minimum of 1 year after discontinuation of study treatment in patients at puberty and sexual maturity.
- •Subject's archival tumour sample (formalin-fixed, paraffinembedded; FFPE) available for evaluation of GD2 expression.
- •Documented disease progression (during or after completion of at least one line treatment) or any subsequent recurrence.
- •GD2 positive tumor assessed by IHC.
- •Age ≥ 2 years and ≤ 21 years.
- •Life expectancy of at least 12 weeks from the time informed consent was signed.
- •Previous systemic anticancer treatment completed ≥ 3 weeks, major surgery ≥ 2 weeks, and radiation therapy ≥ 4 weeks prior to study enrollment.
- •Recovered from adverse effects of prior surgery, radiotherapy, or anti-neoplastic therapy at the discretion of the investigator.
- •Signing of informed consent for trial participation (including for naxitamab treatment) according with current legal regulations.
排除标准
- •Failure to meet any of the inclusion criteria.
- •Requirement, or likely requirement, for corticosteroids at doses >10 mg prednisolone (or equivalent) per day or other immunosuppressive agents.
- •Diagnosis of other malignancies before study inclusion.
- •Planning to become pregnant (while being treated with IT or naxitamab), pregnancy or breastfeeding.
- •Other acute or persistent disorders, behaviors or abnormal laboratory test results, which might increase the risk related to the participation in this clinical trial or to taking the study drug, or which might influence the interpretation of the study results, or which, in the investigator's opinion, disqualify a patient from participating in the trial.
- •Not eligible to IT.
- •Previous treatment with an anti-GD2 antibody.
- •Hypersensitivity to the study drugs or any of their ingredients (covers IT and naxitamab).
- •Simultaneous treatment with other drugs which might interact with naxitamab or IT regimen.
- •Persistent toxicity related to prior therapy, making it impossible to treat with naxitamab.
- •Significant cardiac conduction abnormalities, including known familial prolonged QT syndrome, or screening QTc >480 msec.
- •Symptoms of congestive heart failure or left ventricular ejection fraction <50%.
- •Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and/or chronic oxygen requirement. In addition, room air pulse oximetry < 94% and/or abnormal pulmonary function tests if these assessments are clinically indicated.
结局指标
主要结局
This primary end point has been redacted following transparency rules and protection of confidential information.
This primary end point has been redacted following transparency rules and protection of confidential information.
Safety and tolerability will be assessed based on an analysis of adverse events on all enrolled subjects. These events will be divided according to severity, seriousness, affected organ or system and analyzed for:
Safety and tolerability will be assessed based on an analysis of adverse events on all enrolled subjects. These events will be divided according to severity, seriousness, affected organ or system and analyzed for:
number of serious adverse events (SAE)
number of serious adverse events (SAE)
the number of adverse events (AE), including events of particular importance to the incidence and severity of adverse events occurring during treatment (TEAE) (encoded according to the preferred term and class of organ systems using the Medical Dictionary for Regulatory Activities (MedDRA); these events will be estimated according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v5.0)
the number of adverse events (AE), including events of particular importance to the incidence and severity of adverse events occurring during treatment (TEAE) (encoded according to the preferred term and class of organ systems using the Medical Dictionary for Regulatory Activities (MedDRA); these events will be estimated according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0 (NCI CTCAE v5.0)
the result of a medical examination with the analysis of recorded vital signs
the result of a medical examination with the analysis of recorded vital signs
assessment of laboratory abnormalities according to NCI CTCAE v5.0
assessment of laboratory abnormalities according to NCI CTCAE v5.0
次要结局
- To assess the efficacy of the use of naxitamab in combination with standard IT chemotherapy versus chemotherapy alone:
- EFS (Event-Free Survival) - from randomization to the date of disease progression, recurrence, second malignancy, death or to date of last follow-up for patients without events,
- PFS (Progression-Free Survival) - from randomization to progression of the disease,
- ORR (Overall Response Rate) - defined as the proportion of patients with a best overall response of complete response (CR) or partial response (PR) according WHO criteria,
- OS (Overall Survival) - from randomization to subject's death.
研究者
Klinika Onkologii i Chirurgii Onkologicznej Dzieci i Młodzieży IMID
Scientific
Instytut Matki I Dziecka