Clinical trial NCT02013336: Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors
Official title: Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Recurrent or Refractory Solid Tumors
Ewing Sarcoma
Rhabdomyosarcoma
Neuroblastoma
Osteosarcoma
Keywords: pediatric
MM-398
cyclophosphamide
irinotecan
Lead sponsor: South Plains Oncology Consortium
Sponsor class: NETWORK
First posted: 2013-12-17
Enrollment: 30
Sex: ALL
Minimum age (years): 12
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: To determine the Maximum Tolerated Dose (MTD) of MM-398 in combination with intravenous cyclophosphamide by assessing the occurrence of dose limiting toxicities
timeFrame: 12 months
Secondary outcomes:
measure: Measurement of plasma levels of study drug to determine the pharmacokinetic properties of MM-398 in combination with cyclophosphamide
timeFrame: 12 months
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: University Of Oklahoma Health Sciences Center
state: Oklahoma
city: Dallas
country: United States
facility: UT Southwestern
state: Texas
city: Houston
country: United States
facility: MD Anderson Children's Cancer Hospital
state: Texas
city: Lubbock
country: United States
facility: Texas Tech University Health Sciences Center
state: Texas
city: Milwaukee
country: United States
facility: Midwest Children's Hospital
state: Wisconsin
Study officials:
affiliation: Midwest Children's Hospital
name: Paul Harker-Murray, MD
role: STUDY_CHAIR
References:
citation: Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.