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A Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma

Title A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma
Therapeutic Area Brain Tumors (Pediatric), Neuroblastoma (pediatric), Medulloblastoma
Principal Investigator Michael J. Kelly, MD
Min Age 0 Years
Max Age 21 Years
Gender Both
Contact Erica Heiden
617 636-8885
More Information


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 the FDA is allowing it to be used in research studies like this one. Limited early observations, suggest that nifurtimox may have anti-tumor activity for neuroblastoma and medulloblastoma. 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 common chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells. We will also be collecting information about any side effects that the drug may have.

Study Details

Inclusion Criteria

  • Relapsed or refractory neuroblastoma or medulloblastoma
  • Otherwise normal organ function

Exclusion Criteria

  • No pregnant or nursing females
  • No current chemotherapy or investigational agents
  • No one weighing less than 3.5 kg (8 lbs)

Study Requirements

Participants will need to have exams, tests, and procedures to find out if they can be in the study. This is called “screening.” These exams, tests and procedures are part of regular cancer care and may be done even if the participant does not join the study. If a person is able and willing to participate in this study after the screening is complete, they will receive the study drugs, including nifurtimox for approximately 18 weeks and return for follow up exams for one year after treatment. Nifurtimox will be given three times a day by mouth, every day, for as long as the participant is on this study, unless they have side effects from the drug or the tumor gets worse. On the first 5 consecutive days at the beginning of each cycle participants will also receive the drugs cyclophosphamide and topotecan. These drugs (cyclophosphamide and topotecan) will be given by vein, over about 30-60 minutes each. Participants will be asked to also have extra research-related blood tests done called pharmacokinetic studies (PK). These PK studies are not optional for people who choose to be in the study. The PK studies will involve having additional blood samples drawn at specific intervals. These blood samples will be tested in a laboratory to determine the level of nifurtimox in the blood. Also, the researchers in the main study want to find out more about the cancer, and in addition to the main study, they would like to store participants' tumor cells and bone marrow so that they can study them in the future. This testing is optional for all participants. After participants are finished taking nifurtimox, they will come for a follow-up exam approximately 30 days after stopping the drug.