Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma
Status:
Completed
Trial end date:
2009-06-01
Target enrollment:
Participant gender:
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.