NCT00601003

Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 112 (actual)
• Sponsor: Giselle Sholler · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Neuroblastoma
• Medulloblastoma

Interventions

Type	Name	Description
DRUG	Nifurtimox	30mg/kg/day PO divided into TID dosing q day
DRUG	Cyclophosphamide	250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
DRUG	Topotecan	0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.

Timeline

• Start date: 2008-01-14
• Primary completion: 2020-04-28
• Completion: 2022-10-28
• First posted: 2008-01-25
• Last updated: 2024-08-06
• Results posted: 2022-12-29

Locations

15 sites across 1 country: United States