Last updated on April 2017

Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma


Brief description of study

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 Study 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.

Clinical Study Identifier: NCT00601003

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William Roberts, MD

Rady Children's Hospital
San Diego, CA United States
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Michael Isakoff, MD

Connecticut Children's Hospital
Hartford, CT United States
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Don Eslin, MD

Arnold Palmer Hospital for Children- MD Anderson
Orlando, FL United States
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Randal Wada, MD

Kapiolani Medical Center for Women and Children
Honolulu, HI United States
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Michael Kelly, MD

Tufts Medical Center
Boston, MA United States
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Giselle Sholler, MD

Helen DeVos Children's Hospital
Grand Rapids, MI United States
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Jawhar Rawwas, MD

Children's Hospital and Clinics on Minnesota
Minneapolis, MN United States
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Kathleen Neville, MD

Children's Mercy Hospitals and Clinics
Kansas City, MO United States
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William Ferguson, MD

Cardinal Glennon Children's Medical Center
Saint Louis, MO United States
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Javier Oesterheld, MD

Levine Children's Hospital
Charlotte, NC United States
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Valerie Brown, MD

Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, PA United States
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Jaqueline Kraveka, MD

Medical University of South Carolina
Charleston, SC United States
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Ted Laetsch, MD

Children's Medical Center
Dallas, TX United States
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Peter Zage, MD, PhD

Texas Children's Cancer and Hematology Centers
Houston, TX United States
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Mark Fluchel, MD

Primary Children's Hospital
Salt Lake City, UT United States
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