Why are we doing this research?
This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The goal is to find the highest dose of MM-398 that can be given safely when it is used together with the chemotherapy drug Cyclophosphamide.
- Primary Outcome Measures:
- To determine the Maximum Tolerated Dose (MTD) of MM-398 in combination with intravenous cyclophosphamide by assessing the occurrence of dose limiting toxicities [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Study Type: Interventional
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Who can participate?
AGES ELIGIBLE FOR STUDY: 12 Months to 20 Years
- Histologically or cytologically-confirmed Ewing sarcoma, rhabdomyosarcoma, neuroblastoma, or osteosarcoma
- Disease progression after prior therapy in locally advanced or metastatic setting
- Measurable or evaluable disease based on the Response Evaluation Criteria in Solid Tumors (RECIST v1.1) criteria
- Age 12 months to <21 years
- Adequate bone marrow reserves, hepatic function, and renal function
- Recovered from effects of any prior surgery or cancer therapy
- Patients 18 years or older will provide written consent. A parent or legal guardian of a patient <18 years of age will provide informed consent and patients 11 to 18 years of age will provide written assent or as per participating
- Clinically significant gastrointestinal disorders
- NYHA Class III or IV congestive heart failure, ventricular arrhythmias or uncontrolled blood pressure
- Active infection or unexplained fever
- Known hypersensitivity to any of the components of MM-398 or other liposomal products
- Recent Investigational therapy
- Pregnant or breast feeding; females of child-bearing potential must test negative for pregnancy at the time of enrollment