Single Arm Evaluation of Romiplostim to Prevent Chemotherapy Induced Thrombocytopenia in Patients With Ewing Sarcoma
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 26
- 试验地点
- 3
- 主要终点
- Number of evaluable participants (11 or fewer of 26) that develop CIT during the continuation phase of compressed-interval chemotherapy compared to institutional historical control rate.
研究概览
简要总结
The goal of this clinical trial is to to assess the efficacy of romiplostim as a supportive care measure in patients with a new diagnosis of Ewing sarcoma receiving interval-compressed chemotherapy. The main questions it aims to answer are:
- To demonstrate the efficacy of romiplostim in patients with newly diagnosed Ewing sarcoma, measured specifically as the rate of CIT, defined as a failure to achieve platelet recovery (≥ 75,000/µL post nadir, without transfusion, or a platelet count sufficient to resume chemotherapy per provider and institutional standard) within 7 days of planned chemotherapy cycle start, measured during the continuation phase (cycle 7 to end of cycle 13 or 16, per AEWS0031/AEWS1221, or AEWS1031 respectively) of interval-compressed chemotherapy (every 2 week vincristine/cyclophosphamide +/- doxorubicin and ifosfamide/etoposide chemotherapy) as compared to published institutional historical control rate.
- To determine the safety of incorporation of romiplostim supportive care when given concurrently with Ewing sarcoma therapy.
- To determine the feasibility of incorporation of romiplostim supportive care into upfront Ewing sarcoma regimens.
详细描述
This is a single-arm, multi-center clinical trial to assess the efficacy of romiplostim as a supportive care measure in patients with a new diagnosis of Ewing sarcoma receiving interval-compressed chemotherapy. Patients will be enrolled and started on romiplostim as early as cycle 1 day 1 of their chemotherapy, but no later than 2 weeks from the start of the 5th cycle. If their platelet count is < 200,000/mm3, patients will start romiplostim based on their weight. Dose escalation of romiplostim may occur weekly if platelet count is < 200,000/mm3. Once maximum dose of romiplostim is reached, this dose level will be maintained every 7 days (+/- 2 days).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age: Patients must be >1 year old at the time of study consent.
- •Diagnosis: Patients with a new diagnosis of Ewing sarcoma treated with interval-compressed chemotherapy as per AEWS0031, AEWS1221, or AEWS
- •Informed Consent: All patients and/or their parents or legally authorized representatives must sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
排除标准
- •Marrow disease: Patients with metastatic Ewing sarcoma to the bone marrow are not eligible. Marrow staging is not required for this study but should be performed if clinically indicated.
- •Concomitant therapy, cancer directed: Patients receiving whole lung radiation, >50% of pelvic irradiation, other substantial bone marrow radiation (i.e. ≥ 50% of vertebral marrow space), or patients undergoing pneumonectomy as a component of local control before cycle 14, are not eligible. These therapies are not an exclusion if instituted during or after cycle
- •Concomitant therapy, non-cancer directed:
- •Patients requiring hematopoietic stem cell rescue are not eligible.
- •Previous use of romiplostim, eltrombopag or any other platelet-producing agent is not allowed.
- •Previous therapy for immune thrombocytopenia and related conditions, including rituximab, mycophenolic acid, protracted systemic steroids, and/or IVIG, is prohibited.
- •Treatment with erythropoietin-stimulating agents is prohibited.
- •Patients receiving another investigational drug are not eligible.
- •Patients who are receiving prophylactic dosing of heparin (i.e. enoxaparin) or oral anticoagulants (i.e. rivaroxaban) for thrombosis prevention may be considered for enrollment but will be excluded from secondary aim 'a' analysis (efficacy measured as the median platelet count and transfusion dependency) given shift in transfusion thresholds.
- •Concurrent Illnesses: Patients with a history of or current diagnosis of bone marrow failure, hematologic malignancy, pro-thrombotic condition, or platelet disorder (including immune or heparin induced thrombocytopenia) are not eligible.
- •Patients who in the opinion of the investigator may not be able to comply with the study (including safety monitoring requirements of the study) are not eligible.
研究组 & 干预措施
Romiplostim administration with chemotherapy cycles
Enrolled patients may start romiplostim, as early as cycle 1 day 1; all patients MUST initiate romiplostim no later than 2 weeks from the start of the 5th cycle of chemotherapy. If plt count < 200,000/mm3, patients will start romiplostim based on their weight.
干预措施: Romiplostim (AMG-531) (Drug)
结局指标
主要结局
Number of evaluable participants (11 or fewer of 26) that develop CIT during the continuation phase of compressed-interval chemotherapy compared to institutional historical control rate.
时间窗: 52 weeks
CIT is defined as failure to achieve platelet recovery ((≥ 75,000/µL post nadir, without transfusion, or a platelet count sufficient to resume chemotherapy per provider and institutional standard) within 7 days of planned chemotherapy cycle start, measured during the continuation phase (beyond cycle 6)
Measure adverse events with the addition of romiplostim when given with chemotherapy.
时间窗: 52 weeks
To determine the safety of incorporation of romiplostim supportive care when given concurrently with Ewing sarcoma therapy.
Number of patients able to receive the majority of planned romiplostim doses.
时间窗: 52 weeks
Feasibility will be met if fewer than 9 of 26 enrolled patients fail to receive at least 60% of planned romiplostim doses (excluding doses held for thrombocytosis or post-operatively) from initiation through at least the end of the 13th cycle of chemotherapy or through at least the end of the 16th cycle of chemotherapy.
次要结局
未报告次要终点