Liposomal Muramyl Tripeptide Phosphatidyl Ethanolamine (L-MTP-PE) for High-risk Osteosarcoma
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 205
- 试验地点
- 2
- 主要终点
- Number of participants with adverse events
研究概览
简要总结
The purpose of this study was to collect information regarding the safety and tolerability of mifamurtide (liposomal muramyl tripeptide phosphatidyl ethanolamine; L-MTP-PE).
详细描述
The drug being tested in this study is called mifamurtide (L-MTP-PE; liposomal muramyl tripeptide phosphatidyl ethanolamine). Mifamurtide is being used to treat people with osteosarcoma, a form of cancer.
This was a patient-access study that looked at adverse events, disease progression, and overall survival in study participants.
The study enrolled 205 patients, of whom 204 were treated with mifamurtide intravenously at a dose of 2 mg/m2 twice weekly (at least 3 days apart) for 12 weeks, and then weekly for an additional 24 weeks, for a total of 48 doses in 36 weeks (following surgery for primary or metastatic disease).
This study was conducted in the United States. Participants could receive treatment for up to 9 months. This study was previously mis-categorized as an interventional study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Had signed informed consent/assent. Voluntary participation in the pharmacokinetic portion of the compassionate access protocol was included in the informed consent but not required for compassionate use participation.
- •Had diagnosis of high grade osteosarcoma with relapsed or recurrent disease, locally or metastatic, with disease not completely resectable or who were unable to complete recommended chemotherapy due to toxicity: relapse, recurrence local or metastatic; unable to have standard surgical resection; abbreviated chemotherapy regimen secondary to toxicity (e.g. hypophosphatemia from ifosfamide, cardiotoxicity from doxorubicin, renal dysfunction from methotrexate, ifosfamide, or cisplatin.)
- •Aged 2 ≤ 50 years.
- •Had adequate hematopoietic function as demonstrated by: 1) Absolute Neutrophil Count (ANC) > 750/microL; Hemoglobin (Hb) > 8 g/dL; Platelets > 30,000/microL.
- •Had adequate hepatic function as documented by 1) ALT < 2.5 x upper limit of normal (ULN) for age; 2) total bilirubin ≤ 1.5 x ULN for age.
- •Had adequate renal function as demonstrated by: 1) Creatinine clearance or radioisotope glomerular filtration rate > 70 mL/min/1.73 m^2; OR, 2) Serum creatinine ≤ 2x ULN for age.
- •Had absence of concurrent active acute infection (i.e., afebrile).
- •In females of child bearing potential (not menopausal for 12 months or no previous surgical sterilization), had a negative pregnancy test. All sexually active participants used an effective means of contraception. Such means included oral contraceptives, Lupron Depot, DepoProvera, and condom with diaphragm and spermicidal jelly.
- •Performance status: Lansky 50-100% (≤ 16 years of age); OR, Eastern Cooperative Oncology Group (ECOG) 0-2 or Karnofsky 50-100% (>16 years of age).
排除标准
- •Had chronic use of corticosteroids or other immunosuppressive agents.
- •Was pregnant or breast-feeding.
研究组 & 干预措施
Mifamurtide (L-MTP-PE)
Mifamurtide (L-MTP-PE), intravenous, at a dose of 2 mg/m^2 twice weekly (at least 3 days apart) for 12 weeks, and then weekly for an additional 24 weeks, for a total of 48 doses in 36 weeks.
干预措施: Mifamurtide (L-MTP-PE) (Drug)
结局指标
主要结局
Number of participants with adverse events
时间窗: 12 months or disease progression, whichever occurs first
次要结局
- Serum concentration-time profiles of free and total mifamurtide in 15-20 patients(Just before the start of the first infusion of mifamurtide and at 0.5, 1, 2, 4, 6 and, 24 hours following the start of the first infusion and just prior to the 2nd dose of mifamurtide)
- Overall survival(From date of enrollment to date of death)
- Progression-free survival(From date of enrollment to date of first documented disease progression or death)