Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Neuroblastoma |
Drug: Nifurtimox |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase I Trial of Nifurtimox for Relapsed or Refractory Neuroblastoma |
- Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma alone and in combination with cyclophosphamide and topotecan. [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]
- To evaluate the pharmacokinetic profile of nifurtimox alone and in combination with cyclophosphamide and topotecan. [ Time Frame: 3 months ] [ Designated as safety issue: No ]
- To determine the response rate to treatment with nifurtimox combined with cyclophosphamide/topotecan [ Time Frame: 3 months ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 20 |
| Study Start Date: | November 2006 |
| Study Completion Date: | June 2009 |
| Primary Completion Date: | November 2007 (Final data collection date for primary outcome measure) |
-
Drug: Nifurtimox
Eligibility| Ages Eligible for Study: | up to 21 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age: 0-21 years at the time of study entry.
- Diagnosis: Histologic verification of neuroblastoma at original diagnosis or relapse.
- 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).
- 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).
- Patients must have adequate liver function as defined by AST or ALT <10x normal and a bilirubin <1.5mg/dl
- 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 <3 months
- Investigational Drugs: Patients who are currently receiving another investigational drug.
- Anti-cancer Agents: Patients who are currently receiving other anticancer agents.
- Infection: Patients who have an uncontrolled infection.
- Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.
- Patients may not receive bisphosphonates (i.e. Zometa) within 7 days of start of therapy.
Contacts and Locations| 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 | |
| Study Chair: | Giselle Sholler, MD | University of Vermont / Vermont Children's Hospital |
More Information
Publications:
| Responsible Party: | Giselle Sholler, MD, University of Vermont |
| ClinicalTrials.gov Identifier: | NCT00486564 History of Changes |
| Other Study ID Numbers: | V0610 |
| Study First Received: | June 12, 2007 |
| Last Updated: | October 22, 2009 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by University of Vermont:
|
Treatment Refractory or Relapsed |
Additional relevant MeSH terms:
|
Neuroblastoma 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 Nifurtimox Trypanocidal Agents Antiprotozoal Agents Antiparasitic Agents Anti-Infective Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 19, 2013