Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
NCT ID: NCT00601003
Last Updated: 2024-08-06
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
112 participants
INTERVENTIONAL
2008-01-14
2022-10-28
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Nifurtimox
Nifurtimox
30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide
250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan
0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Interventions
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Nifurtimox
30mg/kg/day PO divided into TID dosing q day
Cyclophosphamide
250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Topotecan
0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
21 Years
ALL
No
Sponsors
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Bayer
INDUSTRY
Giselle Sholler
OTHER
Responsible Party
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Giselle Sholler
Study Chair
Principal Investigators
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Giselle Sholler, MD
Role: STUDY_CHAIR
Beat Childhood Cancer at Atrium Health
Locations
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Rady Children's Hospital
San Diego, California, United States
Connecticut Children's Hospital
Hartford, Connecticut, United States
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States
Tufts Medical Center
Boston, Massachusetts, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Children's Hospital and Clinics on Minnesota
Minneapolis, Minnesota, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, United States
Levine Children's Hospital
Charlotte, North Carolina, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Children's Medical Center
Dallas, Texas, United States
Texas Children's Cancer and Hematology Centers
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Countries
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Document Type: Informed Consent Form
Related Links
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Beat Childhood Cancer Consortium website
Other Identifiers
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V0706
Identifier Type: -
Identifier Source: org_study_id
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