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Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma

NCT00486564 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2009-10-23

No results posted yet for this study

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

Interventions

DRUG

Nifurtimox

Escalating dose by cohort starting at 20mg/kg/day. PO drug taken TID.

Sponsors & Collaborators

  • St. Louis University

    collaborator OTHER

  • University of Vermont

    lead OTHER

Principal Investigators

  • Giselle Sholler, MD · University of Vermont / Vermont Children's Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-11-30
Primary Completion
2007-11-30
Completion
2009-06-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00486564 on ClinicalTrials.gov