NeuroblastomaPhase 1NCT00486564
Safety Study of Nifurtimox for Relapsed or Refractory Neuroblastoma
A Phase 1 study testing Nifurtimox in people with Neuroblastoma, run by University of Vermont at 2 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Nifurtimox
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- Nov 2006to Jun 2009
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 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
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
University of Vermont/Vermont Children's Hospital
Burlington, Vermont, 05401
St. Louis University/Cardinal Glennon Childrens Medical Center
St Louis, Missouri, 63104
About this study
Official title: A Phase I Trial of Nifurtimox for Relapsed or Refractory Neuroblastoma
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.
- Conditions
- Neuroblastoma
- Being tested
- Nifurtimox
- Keywords
- Treatment, Refractory or Relapsed
- Medical subject headings
- Neuroblastoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NifurtimoxDrug
Escalating dose by cohort starting at 20mg/kg/day. PO drug taken TID.
What is being measured
The primary outcome is the main question the study is designed to answer.
Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma alone and in combination with cyclophosphamide and topotecan.
Measured: 3 months
Secondary outcomes (2)
To evaluate the pharmacokinetic profile of nifurtimox alone and in combination with cyclophosphamide and topotecan.
Measured: 3 months
To determine the response rate to treatment with nifurtimox combined with cyclophosphamide/topotecan
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2007 (estimated)
- Study startNov 2006
- Primary completionNov 2007
- Study completionJun 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Vermont
- Sponsor type
- University, hospital or other
- Collaborators
- St. Louis University
- Investigators
- Giselle Sholler, MD, Study chair, University of Vermont / Vermont Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00486564
- Sponsor's protocol ID
- V0610
- Status last verified
- Oct 2009
- First submitted
- 12 Jun 2007
- Last updated
- 23 Oct 2009
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Saulnier Sholler GL, Kalkunte S, Greenlaw C, McCarten K, Forman E. Antitumor activity of nifurtimox observed in a patient with neuroblastoma. J Pediatr Hematol Oncol. 2006 Oct;28(10):693-5. doi: 10.1097/01.mph.0000212994.56812.f2.
PubMed 17023833
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Trial of Nifurtimox for Relapsed or Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT00486564
- Acronym / sponsor ID
- V0610
- Conditions
- Neuroblastoma
- Keywords
- Treatment, Refractory or Relapsed
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of VermontUniversity, hospital or other
- Collaborators
- St. Louis University
- Study start
- Nov 2006
- Primary completion
- Nov 2007 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 14 Jun 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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).
Key exclusion criteria (6 listed)
- 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.
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 |
|---|---|---|
| NifurtimoxEscalating dose by cohort starting at 20mg/kg/day. PO drug taken TID. | Drug | – |
Primary outcome
Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma alone and in combination with cyclophosphamide and topotecan.
Time frame: 3 months
2 secondary outcomes
To evaluate the pharmacokinetic profile of nifurtimox alone and in combination with cyclophosphamide and topotecan.
Time frame: 3 months
To determine the response rate to treatment with nifurtimox combined with cyclophosphamide/topotecan
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2009.
- Background: PubMed 17023833Saulnier Sholler GL, Kalkunte S, Greenlaw C, McCarten K, Forman E. Antitumor activity of nifurtimox observed in a patient with neuroblastoma. J Pediatr Hematol Oncol. 2006 Oct;28(10):693-5. doi: 10.1097/01.mph.0000212994.56812.f2.
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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
University of Vermont / Vermont Children's Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Nifurtimox in people with Neuroblastoma, run by University of Vermont at 2 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 7 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 23 Oct 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Test the safety of nifurtimox in children with relapsed or refractory neuroblastoma alone and in combination with cyclophosphamide and topotecan. (time frame: 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00486564, which also lists 1 linked publication.
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 23 Oct 2009.