Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors

Study Overview
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Brief Summary
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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.

Official Title
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Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors

Conditions
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Recurrent or Refractory Solid Tumors, Ewing Sarcoma, Rhabdomyosarcoma, Neuroblastoma, Osteosarcoma

Intervention / Treatment
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* Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 institutional policy. Exclusion Criteria: 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

Ages Eligible for Study
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Minimum Age: 12 Months
Maximum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MM-398 + cyclophosphamide<br>MM-398+cyclophosphamide | Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide<br>* chemotherapy<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To determine the Maximum Tolerated Dose (MTD) of MM-398 in combination with intravenous cyclophosphamide by assessing the occurrence of dose limiting toxicities |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Measurement of plasma levels of study drug to determine the pharmacokinetic properties of MM-398 in combination with cyclophosphamide |  | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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pediatric, MM-398, cyclophosphamide, irinotecan