Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.
1 other identifier
interventional
112
1 country
15
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Jan 2008
Longer than P75 for phase_2
15 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 14, 2008
CompletedStudy Start
First participant enrolled
January 14, 2008
CompletedFirst Posted
Study publicly available on registry
January 25, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 28, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
October 28, 2022
CompletedResults Posted
Study results publicly available
December 29, 2022
CompletedAugust 6, 2024
August 1, 2024
12.3 years
January 14, 2008
October 28, 2022
August 2, 2024
Conditions
Outcome Measures
Primary Outcomes (2)
Number of Participants With Related Adverse Events as a Measure of Safety and Tolerability
Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma or medulloblastoma in combination with cyclophosphamide/topotecan
2 years
Best Radiological Response in Participants Using the RECIST Criteria
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.
2 years
Study Arms (1)
Nifurtimox
EXPERIMENTALInterventions
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.
Eligibility Criteria
You may qualify if:
- 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.
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Giselle Shollerlead
- Bayercollaborator
Study Sites (15)
Rady Children's Hospital
San Diego, California, 92123, United States
Connecticut Children's Hospital
Hartford, Connecticut, 06106, United States
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, 32806, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813, United States
Tufts Medical Center
Boston, Massachusetts, 02111, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
Children's Hospital and Clinics on Minnesota
Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104, United States
Levine Children's Hospital
Charlotte, North Carolina, 28204, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Children's Medical Center
Dallas, Texas, 75235, United States
Texas Children's Cancer and Hematology Centers
Houston, Texas, 77030, United States
Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Giselle Sholler, MD
- Organization
- Beat Childhood Cancer
Study Officials
- STUDY CHAIR
Giselle Sholler, MD
Beat Childhood Cancer at Atrium Health
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Study Chair
Study Record Dates
First Submitted
January 14, 2008
First Posted
January 25, 2008
Study Start
January 14, 2008
Primary Completion
April 28, 2020
Study Completion
October 28, 2022
Last Updated
August 6, 2024
Results First Posted
December 29, 2022
Record last verified: 2024-08