Clinical Trials /

Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma



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.

Related Conditions:
  • Medulloblastoma
  • Neuroblastoma
Recruiting Status:

Active, not recruiting


Phase 2

Trial Eligibility



  • Brief Title: Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
  • Official Title: A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.

Clinical Trial IDs

  • ORG STUDY ID: V0706
  • NCT ID: NCT00601003


  • Neuroblastoma
  • Medulloblastoma




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.

Detailed Description

      This study is being done to test the effect of a drug, nifurtimox, against neuroblastoma and
      medulloblastoma in children. Nifurtimox is a drug that has been used in South America for
      many years to treat a parasitic disease known as Chagas Disease. It is not approved by the
      Food and Drug Administration for routine use in neuroblastoma or medulloblastoma in the
      United States, but limited early observations suggest that nifurtimox may have anti tumor
      activity for neuroblastoma and medulloblastoma.

      From the preliminary trials of nifurtimox we have determined a safely tolerated dose of
      nifurtimox to use in neuroblastoma patients (30mg/kg/day). The dose determined in the Phase I
      study to be safe, will be the dose used for this study. From clinical experience in South
      America, we know that children can tolerate nifurtimox when given by mouth, and it appears to
      have no long-term side effects when used to treat Chagas Disease. Based on our laboratory and
      animal studies, we believe that drug levels similar to those used to treat Chagas Disease may
      shrink/kill neuroblastoma cells, especially when combined with other chemotherapy drugs. We
      do not know whether nifurtimox will shrink/kill tumor cells effectively in children.
      Therefore, the major goal of the study is to learn if nifurtimox in combination with other
      chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells.

Trial Arms

  • Nifurtimox
  • Cyclophosphamide
  • Topotecan

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

          -  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

        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
Maximum Eligible Age:21 Years
Minimum Eligible Age:N/A
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time Frame:2 years
Safety Issue:
Description:Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan

Secondary Outcome Measures

Measure:Evaluate the correlation between the pharmacologic serum levels of nifurtimox (in combination with cyclophosphamide and topotecan) with tumor response.
Time Frame:2 years
Safety Issue:
Measure:Quality of life and neurocognitive evaluation/questionnaire.
Time Frame:2 years
Safety Issue:
Measure:Biology studies to include: genomic analysis of cells pre- and post- treatment, correlation of in vitro response to in vivo response, flow cytometry of tumor burden in bone marrow and biomarker development.
Time Frame:2 years
Safety Issue:


Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Active, not recruiting
Lead Sponsor:Giselle SaulnierSholler

Last Updated

June 22, 2021