Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
Condition: Neuroblastoma; Medulloblastoma
MeSH terms: Neuroblastoma; Medulloblastoma
Intervention: Nifurtimox; Cyclophosphamide; Topotecan
Intervention description: 30mg/kg/day PO divided into TID dosing q day; 250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.; 0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Intervention MeSH terms: Nifurtimox; Cyclophosphamide; Topotecan
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 112.0
Sex: All
Maximum age: 21 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to determine whether nifurtimox in combination with
      cyclophosphamide and topotecan are effective in the treatment of relapsed or refractory
      neuroblastoma and medulloblastoma.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Age: 0-21 years at the time of diagnosis.

          -  Diagnosis: Histologic verification at either the time of original diagnosis or relapse
             of neuroblastoma or medulloblastoma.

          -  Disease Status: Refractory or first or multiple relapsed neuroblastoma, or
             medulloblastoma that has relapsed after, or is refractory to, a
             chemotherapy-containing treatment regimen.

          -  Measurable disease, including at least one of the following:

               -  Measurable tumor by CT or MRI

               -  For neuroblastoma patients only, a positive MIBG (MIBG not required if subject's
                  neuroblastoma is previously determined to not uptake MIBG), abnormal urinary
                  catecholamine levels, or positive bone marrow biopsy/aspirate.

               -  For medulloblastoma patients only, positive CSF cytology

          -  Current disease state must be one for which there is currently no known curative
             therapy.

          -  A negative urine pregnancy test is required for female participants of child bearing
             potential (≥13 years of age).

          -  Organ Function Requirements Patients without bone marrow metastases must have an ANC >
             500/μl and platelet count >50,000/μl.

          -  Patients must have adequate liver function as defined by AST or ALT <10x normal

          -  Informed Consent: All patients and/or legal guardians must sign informed written
             consent. Assent, when appropriate, will be obtained according to institutional
             guidelines

        Exclusion Criteria:

          -  Life expectancy <2 months or Lansky score <50%

          -  Investigational Drugs: Patients who are currently receiving another investigational
             drug are excluded from participation.

          -  Anti-cancer Agents: Patients who are currently receiving other anticancer agents are
             not eligible. Patients must have fully recovered from the effects of prior
             chemotherapy, generally at least 3 weeks from the most recent administration (6 weeks
             for nitrosoureas).

          -  Infection: Patients who have an uncontrolled infection are not eligible until the
             infection is judged to be well controlled.

          -  Patients who, in the opinion of the investigator, may not be able to comply with the
             safety monitoring requirements of the study, or in whom compliance is likely to be
             suboptimal, should be excluded.

        Compensation for travel related expenses may be available