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Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00486564
Recruitment Status : Completed
First Posted : June 14, 2007
Last Update Posted : October 23, 2009
St. Louis University
Information provided by:
University of Vermont

Brief 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.

Condition or disease Intervention/treatment Phase
Neuroblastoma Drug: Nifurtimox Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Trial of Nifurtimox for Relapsed or Refractory Neuroblastoma
Study Start Date : November 2006
Actual Primary Completion Date : November 2007
Actual Study Completion Date : June 2009

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma
Drug Information available for: Nifurtimox

Intervention Details:
  • Drug: Nifurtimox
    Escalating dose by cohort starting at 20mg/kg/day. PO drug taken TID.

Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00486564

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United States, Missouri
St. Louis University/Cardinal Glennon Childrens Medical Center
St. Louis, Missouri, United States, 63104
United States, Vermont
University of Vermont/Vermont Children's Hospital
Burlington, Vermont, United States, 05401
Sponsors and Collaborators
University of Vermont
St. Louis University
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Study Chair: Giselle Sholler, MD University of Vermont / Vermont Children's Hospital
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Responsible Party: Giselle Sholler, MD, University of Vermont Identifier: NCT00486564    
Other Study ID Numbers: V0610
First Posted: June 14, 2007    Key Record Dates
Last Update Posted: October 23, 2009
Last Verified: October 2009
Keywords provided by University of Vermont:
Refractory or Relapsed
Additional relevant MeSH terms:
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Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Trypanocidal Agents
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents