Clinical trial opens for refractory Neuroblastoma and Medulloblastoma to examine the treatment effectiveness of Nifurtimox on these types of pediatric cancer.
A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma (VCC 0706)
Principle Investigator
Giselle Sholler, M.D.
Pediatric Hematology-Oncology
Vermont Cancer Center at UVM College of Medicine
Vermont Children's Hospital
This study is currently recruiting patients
Rationale
This Phase II trial is designed to evaluate the anti-tumor efficacy of nifurtimox in children with neuroblastoma and medulloblastoma. A Phase I trial of nifurtimox in children with multiply relapsed/refractory neuroblastoma has been completed. The primary objective was to determine the maximum tolerated dose of this medication which was found to be 30mg/kg/day divided in 3 doses.
Purpose
Test the efficacy and safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma, both alone and in combination with cyclophosphamide, topotecan and zoledronic acid.
Targeted Patients
Stratum I - First relapsed neuroblastoma
Stratum II - Multiply relapsed/refractory neuroblastoma
Stratum III - Relapsed/refractory medulloblastoma
Eligibility
1. Age: 0-21 years at the time of study entry.
2. Disease Status: Refractory or first or multiple relapsed neuroblastoma, or medulloblastoma that has relapsed after, or is refractory to, a chemotherapy-containing treatment regimen.
3. Measurable disease, including at least one of the following: Measurable tumor by CT or MRI.
4. 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.
5. Current disease state must be one for which there is currently no known curative therapy.
6. Patients must have adequate liver function as defined by AST or ALT <10x normal
Exclusion Criteria
1. Investigational Drugs: Patients who are currently receiving another investigational drug.
2. Anti-cancer Agents: Patients who are currently receiving other anticancer agents (will need to be stopped prior to study entry).
3. Infection: Patients who have an uncontrolled infection.
4. Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.
Summary of Treatment Plan
Nifurtimox will be administered in three divided doses for 42 days (2 cycles). After completion of these 2 cycles of single-agent nifurtimox, the combination of cyclophosphamide, topotecan and zoledronic acid will be added (Cycles 3-12). Nifurtimox will continue orally three times per day. This 21 day cycle of Nifurtimox, Cyclophosphamide, Topotecan and Zometa will be repeated for a total of 10 times. At completion of 12 cycles of therapy, responding patients may continue to receive nifurtimox as single agent or in combination therapy at treating physician's discretion.
Contact
For information about enrolling in the study, contact:
Genevieve Bergendahl, R.N.
Senior Research Nurse Coordinator
Vermont Cancer Center
HSRF 326C
149 Beaumont Avenue
Burlington, VT 05401
Phone: (802) 656-4511
Fax: (802) 656-2140
Genevieve.Bergendahl@uvm.edu
