NeuroblastomaPhase 2NCT00601003
Study of Nifurtimox to Treat Refractory or Relapsed Neuroblastoma or Medulloblastoma
A Phase 2 study testing Nifurtimox, Cyclophosphamide and Topotecan in people with Neuroblastoma and Medulloblastoma, run by Giselle Sholler at 15 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Neuroblastoma, Medulloblastoma
- What is being tested?
- Nifurtimox, Cyclophosphamide and Topotecan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 112 participants
- Where
- 15 sitesUnited States
- Study dates
- 14 Jan 2008to 28 Oct 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Show all 12Show fewer
- 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
Who cannot join
- 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
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Tufts Medical Center
Boston, Massachusetts, 02111
Medical University of South Carolina
Charleston, South Carolina, 29425
Levine Children's Hospital
Charlotte, North Carolina, 28204
Children's Medical Center
Dallas, Texas, 75235
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Connecticut Children's Hospital
Hartford, Connecticut, 06106
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813
Texas Children's Cancer and Hematology Centers
Houston, Texas, 77030
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
Children's Hospital and Clinics on Minnesota
Minneapolis, Minnesota, 55404
Arnold Palmer Hospital for Children- MD Anderson
Orlando, Florida, 32806
Primary Children's Hospital
Salt Lake City, Utah, 84113
Rady Children's Hospital
San Diego, California, 92123
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104
About this study
Official title: A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.
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.
Read the detailed description
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.
- Conditions
- Neuroblastoma, Medulloblastoma
- Being tested
- Nifurtimox, Cyclophosphamide, Topotecan
- Medical subject headings
- Neuroblastoma, Medulloblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NifurtimoxDrug
Also called Lampit
30mg/kg/day PO divided into TID dosing q day
CyclophosphamideDrug
Also called Cytoxan
250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
TopotecanDrug
Also called Hycamptin
0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle.
| Study group | Role | What they receive |
|---|---|---|
| Nifurtimox | Experimental | Not described Receives: Drug: Nifurtimox; Drug: Cyclophosphamide; Drug: Topotecan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2008 (estimated)
- Study start14 Jan 2008
- Primary completion28 Apr 2020
- Study completion28 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 112 participants (actual)
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- Collaborators
- Bayer
- Responsible party
- Sponsor-investigator, Giselle Sholler, Milton S. Hershey Medical Center
- Investigators
- Giselle Sholler, MD, Study chair, Beat Childhood Cancer at Atrium Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00601003
- Sponsor's protocol ID
- V0706
- Status last verified
- Aug 2024
- First submitted
- 14 Jan 2008
- Last updated
- 6 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial of Nifurtimox for Refractory or Relapsed Neuroblastoma or Medulloblastoma.
- ClinicalTrials.gov ID
- NCT00601003
- Acronym / sponsor ID
- V0706
- Conditions
- Neuroblastoma, Medulloblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 112 participants (actual)
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Collaborators
- Bayer
- Study start
- 14 Jan 2008 (actual)
- Primary completion
- 28 Apr 2020 (actual)
- Study completion
- 28 Oct 2022 (actual)
- First posted
- 25 Jan 2008 (estimated)
- Results
- Posted 29 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Nifurtimox | Experimental | Drug: Nifurtimox, Drug: Cyclophosphamide, Drug: Topotecan |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- 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:
- Current disease state must be one for which there is currently no known curative therapy.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NifurtimoxAlso: Lampit30mg/kg/day PO divided into TID dosing q day | Drug | Nifurtimox |
| CyclophosphamideAlso: Cytoxan250 mg/m2/dose in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle. | Drug | Nifurtimox |
| TopotecanAlso: Hycamptin0.75mg/m2/dose, in normal saline, IV, infused over 30 minutes on days 1-5 of each cycle. | Drug | Nifurtimox |
Primary outcome
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Beat Childhood Cancer Consortium websiteLink provided by the sponsor.
Related trials for Neuroblastoma
RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Active, not recruitingObservational study
Genomic Structural Variation in Cancer Susceptibility
Breast Cancer, Colon Cancer
RecruitingPhase 2
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Metastatic Chondrosarcoma, Locally Advanced Chondrosarcoma
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingPhase 1
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Giselle Sholler, MD study chair
Beat Childhood Cancer at Atrium Health
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Nifurtimox, Cyclophosphamide and Topotecan in people with Neuroblastoma and Medulloblastoma, run by Giselle Sholler at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Related Adverse Events as a Measure of Safety and Tolerability (time frame: 2 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2024.