Skip to main content
OpenTrials
Completed

NCT Number: NCT00486564

Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Neuroblastoma Disease Attributes Neoplasms Neoplasms by Histologic Type Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroectodermal Tumors Neuroectodermal Tumors, Primitive Neuroectodermal Tumors, Primitive, Peripheral Pathologic Processes Pathological Conditions, Signs and Symptoms Recurrence

Age range

Up to 21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

St. Louis University/Cardinal Glennon Childrens Medical Center, St Louis, Missouri, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age: 0-21 years at the time of study entry.
  • Diagnosis: Histologic verification of neuroblastoma at original diagnosis or relapse.
  • Disease Status: Refractory or first or multiple relapsed neuroblastoma with measurable disease by radiographic scan (CT or MRI and MIBG), abnormal urinary catecholamine levels, or positive bone marrow biopsy/aspirate. MIBG not required if subject's neuroblastoma is previously determined to not uptake MIBG isotope (not MIBG avid).
  • Current disease state must be one for which there is currently no known curative therapy.
  • A negative urine pregnancy test is required for female participants of child bearing potential (>13 years of age).
  • Patients must have adequate liver function as defined by AST or ALT <10x normal and a bilirubin <1.5mg/dl
  • Informed Consent: All patients and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines

Exclusion criteria

  • Life expectancy <3 months
  • Investigational Drugs: Patients who are currently receiving another investigational drug.
  • Anti-cancer Agents: Patients who are currently receiving other anticancer agents.
  • Infection: Patients who have an uncontrolled infection.
  • Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.
  • Patients may not receive bisphosphonates (i.e. Zometa) within 7 days of start of therapy.

Treatment and study plan

Nifurtimox

Drug

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

Primary outcomes

  1. Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma alone and in combination with cyclophosphamide and topotecan.

    Time frame: 3 months

Secondary outcomes

  1. To evaluate the pharmacokinetic profile of nifurtimox alone and in combination with cyclophosphamide and topotecan.

    Time frame: 3 months

  2. To determine the response rate to treatment with nifurtimox combined with cyclophosphamide/topotecan

    Time frame: 3 months

Sponsors and collaborators

Lead sponsor

University of Vermont

Other

Collaborators

  • St. Louis University

Registry information

Official study title

A Phase I Trial of Nifurtimox for Relapsed or Refractory Neuroblastoma

Important dates

Study start
2006
Primary completion
2007
Study completion
2009
First posted
Jun 14, 2007
Registry last updated
Oct 23, 2009

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.