Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma

October 22, 2009 updated by: University of Vermont

A Phase I Trial 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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Missouri
      • St. Louis, Missouri, United States, 63104
        • St. Louis University/Cardinal Glennon Childrens Medical Center
    • Vermont
      • Burlington, Vermont, United States, 05401
        • University of Vermont/Vermont Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Age: 0-21 years at the time of study entry.
  2. Diagnosis: Histologic verification of neuroblastoma at original diagnosis or relapse.
  3. 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).
  4. Current disease state must be one for which there is currently no known curative therapy.
  5. A negative urine pregnancy test is required for female participants of child bearing potential (>13 years of age).
  6. Patients must have adequate liver function as defined by AST or ALT <10x normal and a bilirubin <1.5mg/dl
  7. 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:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma alone and in combination with cyclophosphamide and topotecan.
Time Frame: 3 months
3 months

Secondary Outcome Measures

Outcome Measure
Time Frame
To evaluate the pharmacokinetic profile of nifurtimox alone and in combination with cyclophosphamide and topotecan.
Time Frame: 3 months
3 months
To determine the response rate to treatment with nifurtimox combined with cyclophosphamide/topotecan
Time Frame: 3 months
3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Vermont

Collaborators

St. Louis University

Investigators

  • Study Chair: Giselle Sholler, MD, University of Vermont / Vermont Children's Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2006

Primary Completion (Actual)

November 1, 2007

Study Completion (Actual)

June 1, 2009

Study Registration Dates

First Submitted

June 12, 2007

First Submitted That Met QC Criteria

June 13, 2007

First Posted (Estimate)

June 14, 2007

Study Record Updates

Last Update Posted (Estimate)

October 23, 2009

Last Update Submitted That Met QC Criteria

October 22, 2009

Last Verified

October 1, 2009

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • V0610

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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