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.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan
2 years
Yes
Giselle Sholler, MD
Study Chair
Van Andle Research Institute
United States: Food and Drug Administration
V0706
NCT00601003
February 2008
January 2018
Name | Location |
---|---|
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 |