Trial record 1 of 1 for:    nifurtimox and medulloblastoma
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Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by Spectrum Health Hospitals
Sponsor:
Collaborator:
Bayer
Information provided by (Responsible Party):
Giselle Sholler, Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT00601003
First received: January 14, 2008
Last updated: February 5, 2014
Last verified: February 2014

January 14, 2008
February 5, 2014
February 2008
December 2014   (final data collection date for primary outcome measure)
  • Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 2 years ] [ Designated as safety issue: Yes ]
    Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan
  • Best Radiological Response in Participants using the RECIST criteria [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Test the efficacy of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan
Test the efficacy and safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma, both alone and in combination with cyclophosphamide/topotecan, plus—in patients with bone metastases—zoledronic acid. [ Time Frame: Ongoing ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00601003 on ClinicalTrials.gov Archive Site
  • Evaluate the correlation between the pharmacologic serum levels of nifurtimox (in combination with cyclophosphamide and topotecan) with tumor response. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Quality of life and neurocognitive evaluation/questionnaire. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Biology studies to include: genomic analysis of cells pre- and post- treatment, correlation of in vitro response to in vivo response, flow cytometry of tumor burden in bone marrow and biomarker development. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
  • Evaluate the correlation between the pharmacologic serum levels of nifurtimox (given alone and in combination with cyclophosphamide and topotecan) with tumor response. [ Time Frame: Ongoing ] [ Designated as safety issue: No ]
  • Quality of life and neurocognitive evaluation/questionnaire. [ Time Frame: Ongoing ] [ Designated as safety issue: No ]
  • Biology studies to include: genomic analysis of cells pre- and post- treatment, correlation of in vitro response to in vivo response, flow cytometry of tumor burden in bone marrow and biomarker development. [ Time Frame: Ongoing ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.

The purpose of this study is to determine whether nifurtimox in combination with cyclophosphamide and topotecan are effective in the treatment of relapsed or refractory neuroblastoma and medulloblastoma.

This study is being done to test the effect of a drug, nifurtimox, against neuroblastoma and medulloblastoma in children. Nifurtimox is a drug that has been used in South America for many years to treat a parasitic disease known as Chagas Disease. It is not approved by the Food and Drug Administration for routine use in neuroblastoma or medulloblastoma in the United States, but limited early observations suggest that nifurtimox may have anti tumor activity for neuroblastoma and medulloblastoma.

From the preliminary trials of nifurtimox we have determined a safely tolerated dose of nifurtimox to use in neuroblastoma patients (30mg/kg/day). The dose determined in the Phase I study to be safe, will be the dose used for this study. From clinical experience in South America, we know that children can tolerate nifurtimox when given by mouth, and it appears to have no long-term side effects when used to treat Chagas Disease. Based on our laboratory and animal studies, we believe that drug levels similar to those used to treat Chagas Disease may shrink/kill neuroblastoma cells, especially when combined with other chemotherapy drugs. We do not know whether nifurtimox will shrink/kill tumor cells effectively in children. Therefore, the major goal of the study is to learn if nifurtimox in combination with other chemotherapy drugs is effective in shrinking/killing neuroblastoma and medulloblastoma cells.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Neuroblastoma
  • Medulloblastoma
  • Drug: Nifurtimox
    30mg/kg/day PO divided into TID dosing q day
    Other Name: Lampit
  • Drug: Cyclophosphamide
    250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
    Other Name: Cytoxan
  • Drug: Topotecan
    0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
    Other Name: Hycamptin
Experimental: Nifurtimox
Interventions:
  • Drug: Nifurtimox
  • Drug: Cyclophosphamide
  • Drug: Topotecan
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
100
December 2018
December 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Age: 0-21 years at the time of diagnosis.
  • Diagnosis: Histologic verification at either the time of original diagnosis or relapse of neuroblastoma or medulloblastoma.
  • Disease Status: Refractory or first or multiple relapsed neuroblastoma, or medulloblastoma that has relapsed after, or is refractory to, a chemotherapy-containing treatment regimen.
  • Measurable disease, including at least one of the following:

    • Measurable tumor by CT or MRI
    • For neuroblastoma patients only, a positive MIBG (MIBG not required if subject's neuroblastoma is previously determined to not uptake MIBG), abnormal urinary catecholamine levels, or positive bone marrow biopsy/aspirate.
    • For medulloblastoma patients only, positive CSF cytology
  • 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).
  • Organ Function Requirements Patients without bone marrow metastases must have an ANC > 500/μl and platelet count >50,000/μl.
  • Patients must have adequate liver function as defined by AST or ALT <10x normal
  • 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 <2 months or Lansky score <50%
  • Investigational Drugs: Patients who are currently receiving another investigational drug are excluded from participation.
  • Anti-cancer Agents: Patients who are currently receiving other anticancer agents are not eligible. Patients must have fully recovered from the effects of prior chemotherapy, generally at least 3 weeks from the most recent administration (6 weeks for nitrosoureas).
  • Infection: Patients who have an uncontrolled infection are not eligible until the infection is judged to be well controlled.
  • Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.

Compensation for travel related expenses may be available

Both
up to 21 Years
No
Contact: Genevieve Bergendahl, RN 616-267-0335 genevieve.bergendahl@helendevoschildrens.org
Contact: Alyssa VanderWerff (616) 267-0327 alyssa.vanderwerff@helendevoschildrens.org
United States
 
NCT00601003
V0706
Yes
Giselle Sholler, Spectrum Health Hospitals
Giselle Sholler
Bayer
Study Chair: Giselle Sholler, MD The Spectrum Health Group
Spectrum Health Hospitals
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP