Neoplasms

All Conditions

Rare Diseases

Medulloblastoma

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Glioma

Neuroepithelioma

Antineoplastic Agents

Antirheumatic Agents

All Drugs and Chemicals

Anti-Infective Agents

Cyclophosphamide

Topotecan

Nifurtimox

Immunosuppressive Agents

Immunologic Factors

Antineoplastic Agents, Alkylating

Alkylating Agents

Topoisomerase I Inhibitors

Enzyme Inhibitors

Antiprotozoal Agents

Antiparasitic Agents

30mg/kg/day PO divided into TID dosing q day

250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.

0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.

Giselle Sholler, MD

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.

Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma

A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.

Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan

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.

Bayer

Giselle Sholler

Study Chair

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.

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Number of subjects that signed consent and had baseline information collected

Number of Participants With Related Adverse Events as a Measure of Safety and Tolerability

Best Radiological Response in Participants Using the RECIST Criteria

Overall Study

Escalating dose by cohort starting at 20mg/kg/day. PO drug taken TID.

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.

Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma

A Phase I Trial of Nifurtimox for Relapsed or Refractory Neuroblastoma

St. Louis University

University of Vermont

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma	NCT00601003	Neuroblastoma Medulloblastoma	Nifurtimox Cyclophosphamid... Topotecan	- 21 Years	Milton S. Hershey Medical Center
Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma	NCT00601003	Neuroblastoma Medulloblastoma	Nifurtimox Cyclophosphamid... Topotecan	- 21 Years	Milton S. Hershey Medical Center
Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma	NCT00486564	Neuroblastoma	Nifurtimox	- 21 Years	University of Vermont

Spots Global Cancer Trial Database for nifurtimox

Every month we try and update this database with for nifurtimox cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for nifurtimox

Every month we try and update this database with for nifurtimox cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for nifurtimox cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.