Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
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| ClinicalTrials.gov Identifier: NCT00601003 |
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Recruitment Status :
Completed
First Posted : January 25, 2008
Results First Posted : December 29, 2022
Last Update Posted : January 4, 2023
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Neuroblastoma Medulloblastoma | Drug: Nifurtimox Drug: Cyclophosphamide Drug: Topotecan | Phase 2 |
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.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 112 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma. |
| Actual Study Start Date : | January 14, 2008 |
| Actual Primary Completion Date : | April 28, 2020 |
| Actual Study Completion Date : | October 28, 2022 |
| Arm | Intervention/treatment |
|---|---|
| Experimental: Nifurtimox |
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 |
- Number of Participants With Related Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 2 years ]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 ]
Test the efficacy of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions, Overall Best Response assessed by CT or MRI, MIBG, and Bone Marrow: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions, bone marrow with CR, and MIBG with CR/PR. Overall Response (OR) = CR + PR.
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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:
- 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.
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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
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 ClinicalTrials.gov identifier (NCT number): NCT00601003
| United States, California | |
| Rady Children's Hospital | |
| San Diego, California, United States, 92123 | |
| United States, Connecticut | |
| Connecticut Children's Hospital | |
| Hartford, Connecticut, United States, 06106 | |
| United States, Florida | |
| Arnold Palmer Hospital for Children- MD Anderson | |
| Orlando, Florida, United States, 32806 | |
| United States, Hawaii | |
| Kapiolani Medical Center for Women and Children | |
| Honolulu, Hawaii, United States, 96813 | |
| United States, Massachusetts | |
| Tufts Medical Center | |
| Boston, Massachusetts, United States, 02111 | |
| United States, Michigan | |
| Helen DeVos Children's Hospital | |
| Grand Rapids, Michigan, United States, 49503 | |
| United States, Minnesota | |
| Children's Hospital and Clinics on Minnesota | |
| Minneapolis, Minnesota, United States, 55404 | |
| United States, Missouri | |
| Children's Mercy Hospitals and Clinics | |
| Kansas City, Missouri, United States, 64108 | |
| Cardinal Glennon Children's Medical Center | |
| Saint Louis, Missouri, United States, 63104 | |
| United States, North Carolina | |
| Levine Children's Hospital | |
| Charlotte, North Carolina, United States, 28204 | |
| United States, Pennsylvania | |
| Penn State Milton S. Hershey Medical Center and Children's Hospital | |
| Hershey, Pennsylvania, United States, 17033 | |
| United States, South Carolina | |
| Medical University of South Carolina | |
| Charleston, South Carolina, United States, 29425 | |
| United States, Texas | |
| Children's Medical Center | |
| Dallas, Texas, United States, 75235 | |
| Texas Children's Cancer and Hematology Centers | |
| Houston, Texas, United States, 77030 | |
| United States, Utah | |
| Primary Children's Hospital | |
| Salt Lake City, Utah, United States, 84113 | |
| Study Chair: | Giselle Sholler, MD | Beat Childhood Cancer at Atrium Health |
Documents provided by Wake Forest University Health Sciences:
| Responsible Party: | Wake Forest University Health Sciences |
| ClinicalTrials.gov Identifier: | NCT00601003 |
| Other Study ID Numbers: |
V0706 |
| First Posted: | January 25, 2008 Key Record Dates |
| Results First Posted: | December 29, 2022 |
| Last Update Posted: | January 4, 2023 |
| Last Verified: | November 2022 |
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Neuroblastoma Medulloblastoma Neuroectodermal Tumors, Primitive, Peripheral Neuroectodermal Tumors, Primitive Neoplasms, Neuroepithelial Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal Neoplasms by Histologic Type Neoplasms Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Glioma Nifurtimox Cyclophosphamide Topotecan |
Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Antirheumatic Agents Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Topoisomerase I Inhibitors Topoisomerase Inhibitors Enzyme Inhibitors Trypanocidal Agents Antiprotozoal Agents Antiparasitic Agents |