NCT00486564

Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma

A Phase I Trial of Nifurtimox for Relapsed or Refractory Neuroblastoma

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: University of Vermont · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

There is currently no curative treatment for children with relapsed/refractory neuroblastoma, and for these children the 5 year survival rate is \<10%. As such, new therapeutic approaches are needed to treat these children. This Phase 1 clinical trial is specifically designed to test the safety and toxicity of nifurtimox when given in combination with cyclophosphamide and topotecan for the treatment of relapsed and/or refractory neuroblastoma . Prior to study opening, 3 pediatric patients with neuroblastoma have received nifurtimox in combination with this chemotherapy regimen, and all have had significant measurable responses without undue toxicity. These case reports, as well as our in vitro and in vivo investigations into the biologic effect of nifurtimox on neuroblastoma cells has prompted the development of this Phase I study. This Phase I study will involve a dose escalation trial of daily oral nifurtimox alone for one 21 day cycle of therapy, followed by continuation of nifurtimox with the addition of standard doses of cyclophosphamide (5 days) and topotecan (5 days) for 3 additional 21 day cycles. Our primary aim is to evaluate the safety of nifurtimox alone and in combination with these chemotherapy agents in multiply relapsed/refractory patients. Our secondary aim will be to evaluate the pharmacokinetics of nifurtimox as well as treatment response.

Conditions

• Neuroblastoma

Interventions

Type	Name	Description
DRUG	Nifurtimox	Escalating dose by cohort starting at 20mg/kg/day. PO drug taken TID.

Timeline

• Start date: 2006-11-01
• Primary completion: 2007-11-01
• Completion: 2009-06-01
• First posted: 2007-06-14
• Last updated: 2009-10-23

Locations

2 sites across 1 country: United States