High-Risk NeuroblastomaNCT04501757
Naxitamab and GM-CSF in People With Neuroblastoma
A study testing Naxitamab/GM-CSF in people with High-Risk Neuroblastoma, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial summary
This is an Expanded Access Program (EAP) that will give the participants access to the drug naxitamab before it is approved by the FDA. Naxitamab will be combined with granulocyte-macrophage colony stimulating factor (GM-CSF). Read more about this study
- Who is it for?
- Ages 18 months and older
- Conditions the study is for
- High-Risk Neuroblastoma
- What is being tested?
- Naxitamab/GM-CSF
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 18 months and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Patients who are ineligible to participate in study NCT03363373 (IRB# 18-375), will be considered for treatment under this expanded access protocol.
- Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels
- Patients must have HR-NB (MYCN-amplified stage 2/3/4/4S of any age and MYCNnonamplified stage 4 in patients greater than 18 months of age).
- Patients must be in first or greater CR or have refractory or relapsed disease
- If previously treated with any anti-GD2 mAb, human anti-hu3F8 antibody (HAHA) titer must be negative.
Who cannot join
- Active life-threatening infection
- Pregnant women or women who are breastfeeding
- HAHA titer positive
- History of anaphylaxis CTCAE grade 4 to naxitamab
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: Expanded Access Use of Naxitamab/GM-CSF Immunotherapy for Consolidation of Complete Remission or Relapsed/Refractory High-Risk Neuroblastoma
This is an Expanded Access Program (EAP) that will give the participants access to the drug naxitamab before it is approved by the FDA. Naxitamab will be combined with granulocyte-macrophage colony stimulating factor (GM-CSF). Participants in this study will have high-risk neuroblastoma that either went away completely after treatment (complete remission) or has come back (relapsed/refractory).
Researchers think the combination of naxitamab and GM-CSF will be effective because naxitamab and GM-CSF strengthen the immune system's response to cancer cells in different ways. Naxitamab is an antibody, like the proteins made by the immune system to protect the body from harm. Naxitamab helps the cells of the immune system to find and attack cancer cells. GM-CSF is a protein that strengthens the immune system by increasing the number of immune cells called granulocytes. Granulocytes are white blood cells that fight off cancer cells. The combination of naxitamab and GM-CSF is a type of immunotherapy.
- Conditions
- High-Risk Neuroblastoma
- Being tested
- Naxitamab/GM-CSF
- Keywords
- Remission High-Risk Neuroblastoma, Relapsed/Refractory High-Risk Neuroblastoma, Naxitamab, GM-CSF, 20-227
- Medical subject headings
- Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Naxitamab/GM-CSFBiological
naxitamab/GM-CSF for 5 Cycles
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High-Risk NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Brian Kushner, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04501757
- Sponsor's protocol ID
- 20-227
- Status last verified
- Dec 2022
- First submitted
- 30 Jul 2020
- Last updated
- 8 Dec 2022
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Use of Naxitamab/GM-CSF Immunotherapy for Consolidation of Complete Remission or Relapsed/Refractory High-Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT04501757
- Acronym / sponsor ID
- 20-227
- Conditions
- High-Risk Neuroblastoma
- Keywords
- Remission High-Risk Neuroblastoma, Relapsed/Refractory High-Risk Neuroblastoma, Naxitamab, GM-CSF, 20-227
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- First posted
- 6 Aug 2020 (actual)
- 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.
This is an Expanded Access Program (EAP) that will give the participants access to the drug naxitamab before it is approved by the FDA. Naxitamab will be combined with granulocyte-macrophage colony stimulating factor (GM-CSF). Participants in this study will have high-risk neuroblastoma that either went away completely after treatment (complete remission) or has come back (relapsed/refractory).
Researchers think the combination of naxitamab and GM-CSF will be effective because naxitamab and GM-CSF strengthen the immune system's response to cancer cells in different ways. Naxitamab is an antibody, like the proteins made by the immune system to protect the body from harm. Naxitamab helps the cells of the immune system to find and attack cancer cells. GM-CSF is a protein that strengthens the immune system by increasing the number of immune cells called granulocytes. Granulocytes are white blood cells that fight off cancer cells. The combination of naxitamab and GM-CSF is a type of immunotherapy.
Study design
Who may qualify
- Ages
- 18 months and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients who are ineligible to participate in study NCT03363373 (IRB# 18-375), will be considered for treatment under this expanded access protocol.
- Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels
- Patients must have HR-NB (MYCN-amplified stage 2/3/4/4S of any age and MYCNnonamplified stage 4 in patients greater than 18 months of age).
- Patients must be in first or greater CR or have refractory or relapsed disease
- If previously treated with any anti-GD2 mAb, human anti-hu3F8 antibody (HAHA) titer must be negative.
Key exclusion criteria (4 listed)
- Active life-threatening infection
- Pregnant women or women who are breastfeeding
- HAHA titer positive
- History of anaphylaxis CTCAE grade 4 to naxitamab
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 |
|---|---|---|
| Naxitamab/GM-CSFnaxitamab/GM-CSF for 5 Cycles | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2022.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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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.
Brian Kushner, MD principal investigator
Memorial Sloan Kettering Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Naxitamab/GM-CSF in people with High-Risk Neuroblastoma, run by Memorial Sloan Kettering Cancer Center at 1 site 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 5 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 8 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
Are results available for this study?
Results are not available in the source record.
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.
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 8 Dec 2022.