Know Cancer

forgot password

A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.

Phase 2
21 Years
Open (Enrolling)
Neuroblastoma, Medulloblastoma

Thank you

Trial Information

A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.

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.

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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

Outcome Description:

Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan

Outcome Time Frame:

2 years

Safety Issue:


Principal Investigator

Giselle Sholler, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Van Andle Research Institute


United States: Food and Drug Administration

Study ID:




Start Date:

February 2008

Completion Date:

January 2018

Related Keywords:

  • Neuroblastoma
  • Medulloblastoma
  • Medulloblastoma
  • Neuroblastoma



Medical University of South Carolina Charleston, South Carolina  29425-0721
Children's Mercy Hospitals and Clinics Kansas City, Missouri  64108
Rady Children's Hospital San Diego, California  92123
Connecticut Children's Hospital Hartford, Connecticut  06106
Arnold Palmer Hospital for Children- MD Anderson Orlando, Florida  32806
Helen DeVos Children's Hospital Grand Rapids, Michigan  49503
Cardinal Glennon Children's Medical Center St. Louis, Missouri  63104
Levine Children's Hospital Charlotte, North Carolina  28204
Texas Children's Cancer and Hematology Centers Houston, Texas  77030