DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Feasibility of Administering DFMO
研究概览
简要总结
The purpose of this study is to determine the feasibility of administering DL-alpha-difluoromethylornithine (DFMO) to patients with relapsed Ewing sarcoma and osteosarcoma who have completed all planned therapy and have no evidence of disease.
详细描述
Approximately 30-35% of patients diagnosed with osteosarcoma or Ewing sarcoma will develop relapsed/refractory disease and carry a very poor prognosis. DL-alpha-difluoromethylornithine, commonly known as DFMO or eflornithine, is a synthetic analog of the amino acid ornithine. DFMO has been studied in a number of different cancers as either a therapeutic or a chemopreventative agent and is now FDA approved to reduce the risk of relapse in patients with newly diagnosed high-risk neuroblastoma. As DFMO has now been given to over 100 children with metastatic cancer, dosing and safety in this population is well established. Given the stagnant survival rates for children, adolescents, and young adults with relapsed Ewing sarcoma and osteosarcoma over the past few decades, this study will explore the feasibility of using DFMO in patients with relapsed osteosarcoma and relapsed Ewing sarcoma who are without any evidence of disease at the end of therapy in order to prevent disease recurrence.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 39 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients < 40 years of age at the time of enrollment
- •Diagnosis of relapsed osteosarcoma or relapsed Ewing sarcoma who have completed all planned therapy for their relapse, as described in the protocol, and have no evidence of disease
- •Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2
- •Myelosuppressive chemotherapy: At least 14 days must have elapsed since completion of myelosuppressive therapy
- •Monoclonal antibodies: At least 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to Grade < 2
- •Biologic therapy (defined as anti-cancer agents not known to be myelosuppressive): At least 7 days after the last dose of agent
- •Radiation therapy: At least 14 days must have elapsed after local External Beam Radiation Therapy (XRT), at least 90 days after Total Body Irradiation (TBI), craniospinal XRT or if radiation to greater than 50% of the pelvis, and at least 42 days if other substantial bone marrow radiation
- •Adequate bone marrow function defined as:
- •Peripheral absolute neutrophil count (ANC) greater or equal to 750/microliter
- •Platelet count greater or equal to 75,000/microliter (transfusion independent)
- •Adequate renal function defined by serum creatinine based on age and gender (see protocol)
- •Adequate liver function defined as:
- •Total bilirubin ≤ 1.5 x the upper limit of normal (ULN) for age AND
- •SGPT (ALT) ≤ 5.0 x ULN for age. For this study the ULN is 45 U/L
排除标准
- •Pregnant or breastfeeding females. Men and women of childbearing potential and their partners must agree to use adequate contraception while enrolled on this study. Based on the teratogenic potential of the agent, pregnant women will be excluded from this study. Because of potential risks to breastfed infants due to drug metabolites that could be excreted in breast milk, female patients who are lactating must agree to stop breastfeeding or will otherwise be excluded from this study. Females of childbearing potential must have a negative pregnancy test to be eligible for this study
- •Patients must not have an uncontrolled infection
- •Patients with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of study agents or to significantly increase the severity of the toxicities experienced from study treatment are not eligible
研究组 & 干预措施
Treatment with DFMO
DFMO will be administered orally every 12 hours in 28-day cycles at the FDA approved dosages based on the patient's body surface area (BSA). DFMO tablets are 192 mg.
- Patients with a BSA < 1.5 m^2 will take 768 mg (four tablets) orally twice a day.
- Patients with a BSA 0.75 to 1.5 m^2 will take 576 mg (three tablets) orally twice a day.
- Patients with a BSA of 0.5 to < 0.75 m^2 will take 384 mg (two tablets) orally twice a day.
- Patients with a BSA of 0.25 to < 0.5 m^2 will take 192 mg (one tablet) orally twice a day.
干预措施: DFMO (Drug)
结局指标
主要结局
Feasibility of Administering DFMO
时间窗: Up to 2 years
Feasibility will be defined as the ability to successfully administer DFMO to at least 80% of subjects who initiate therapy until either disease recurrence or completion of the maximally allowed duration of therapy. Results will be summarized using basic descriptive statistics.
次要结局
- Event Free Survival(Up to 2 years)