Recurrent or Refractory Solid TumorsPhase 1NCT02013336
Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors
A Phase 1 study testing MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide in people with Recurrent or Refractory Solid Tumors and Ewing Sarcoma, run by South Plains Oncology Consortium at 5 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 months to 20 years
- Conditions the study is for
- Recurrent or Refractory Solid Tumors, Ewing Sarcoma, Rhabdomyosarcoma
- What is being tested?
- MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 5 sitesUnited States
- Study dates
- Dec 2013to Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 months to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
513-636-4200
Joseph Pressey, MD
- Recruiting
UT Southwestern
Dallas, Texas, 75235
Alison Patterson
- Recruiting
Texas Tech University Health Sciences Center
Lubbock, Texas, 79430
Liza Villarreal, 806-775-8590, liza.villarreal@ttuhsc.edu
Erin K Barr, MD
- Recruiting
Midwest Children's Hospital
Milwaukee, Wisconsin, 53226
Sophia Root, 414-955-5025, sroot@mcw.edu
- Recruiting
University Of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Haley Jones
About this study
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.
- Conditions
- Recurrent or Refractory Solid Tumors, Ewing Sarcoma, Rhabdomyosarcoma, Neuroblastoma, Osteosarcoma
- Being tested
- MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide
- Keywords
- pediatric, MM-398, cyclophosphamide, irinotecan
- Medical subject headings
- Recurrence, Sarcoma, Ewing, Rhabdomyosarcoma, Neuroblastoma, Osteosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamideDrug
chemotherapy
| Study group | Role | What they receive |
|---|---|---|
| MM-398 + cyclophosphamide | Experimental | MM-398+cyclophosphamide Receives: Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the Maximum Tolerated Dose (MTD) of MM-398 in combination with intravenous cyclophosphamide by assessing the occurrence of dose limiting toxicities
Measured: 12 months
Secondary outcomes (1)
Measurement of plasma levels of study drug to determine the pharmacokinetic properties of MM-398 in combination with cyclophosphamide
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2013 (estimated)
- Study startDec 2013
- Primary completionDec 2026 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Recurrent or Refractory Solid TumorsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- South Plains Oncology Consortium
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Paul Harker-Murray, MD, Study chair, Midwest Children's HospitalErin K Barr, MD, Study chair, Texas Tech University Health Sciences Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02013336
- Sponsor's protocol ID
- SPOC-2012-001
- Status last verified
- Oct 2026
- First submitted
- 11 Dec 2013
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 31401903
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors
- ClinicalTrials.gov ID
- NCT02013336
- Acronym / sponsor ID
- SPOC-2012-001
- Conditions
- Recurrent or Refractory Solid Tumors, Ewing Sarcoma, Rhabdomyosarcoma, Neuroblastoma, Osteosarcoma
- Keywords
- pediatric, MM-398, cyclophosphamide, irinotecan
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- South Plains Oncology ConsortiumResearch network
- Study start
- Dec 2013 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 17 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MM-398 + cyclophosphamide | Experimental | Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamideMM-398+cyclophosphamide |
Who may qualify
- Ages
- 12 months to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamidechemotherapy | Drug | MM-398 + cyclophosphamide |
Primary outcome
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
1 secondary outcome
Measurement of plasma levels of study drug to determine the pharmacokinetic properties of MM-398 in combination with cyclophosphamide
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
- Derived from this study: PubMed 31401903Hattinger 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.
- Individual participant dataSharing planned: Undecided
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RecruitingPhase 1
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Paul Harker-Murray, MD study chair
Midwest Children's Hospital
Erin K Barr, MD study chair
Texas Tech University Health Sciences Center
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide in people with Recurrent or Refractory Solid Tumors and Ewing Sarcoma, run by South Plains Oncology Consortium at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Amanda Knight, RN, BSN (806-743-2690, amanda.knight@ttuhsc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02013336, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2026.