Olfactory NeuroblastomaNCT04755205
A Natural History Study of Children and Adults With Olfactory Neuroblastoma
An observational study of Olfactory Neuroblastoma and Esthesioneuroblastoma, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: Olfactory neuroblastoma (ONB) is a rare cancer. It grows from tissue in the upper part of the nose cavity, related to the sense of smell and can affect a person s sense of smell. Researchers want to better understand the health problems of people with ONB. Read more about this study
- Who is it for?
- Ages 3 to 120 years
- Conditions the study is for
- Olfactory Neuroblastoma, Esthesioneuroblastoma
- How many people
- 525 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jun 2022to 31 Dec 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 3 to 120 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with olfactory neuroblastoma.
Who may be able to join
- Subjects with histologically documented olfactory neuroblastoma.
- Age >= 3 years old.
- Ability of subject to understand and the willingness to sign a written consent document
Who cannot join
- Pregnant women are excluded from this study because of more than minimal risk activities (imaging studies with contrast, biopsies) pertaining to enrollment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
For more information at the NIH Clinical Center contact National Cancer Institute Referral Office, 888-624-1937
About this study
Background:
Olfactory neuroblastoma (ONB) is a rare cancer. It grows from tissue in the upper part of the nose cavity, related to the sense of smell and can affect a person s sense of smell. Researchers want to better understand the health problems of people with ONB. This may help them design better treatment and supportive care studies.
Read more
Objective:
To better understand ONB-the course of the disease, tumor characteristics, response to treatments, and management of the treatment.
Eligibility:
People ages 3 years and older who have ONB. They must enroll in NIH studies #19-C-0016 and #18-DC-0051.
Design:
Participants will be screened with a medical history and medical record review.
Participants do not have to visit NIH.
Participants will give a blood sample. They will complete surveys to assess their emotional and physical wellbeing and needs. Leftover tissue from biopsies and surgeries will be collected.
Participants will take smell tests. They will smell items and answer questions about them.
Participants may take taste tests. They will get plastic taste strips that they will move around their mouth to determine the taste.
Participants may have a physical exam. Their performance status may be assessed.
Participants may give blood, saliva, urine, and nasal secretion samples.
Participants may have computed tomography and/or magnetic resonance imaging scans.
Participants may have one or more tumor biopsies.
Participants will talk to the research team about the results of their medical record/tests evaluation. The team will recommend how to best manage and treat their disease.
Participants may give samples and complete surveys every 12 months. Their medical records will be reviewed every year. They will be monitored for the rest of their life.
Read the detailed description
Background:
Olfactory neuroblastoma (ONB), also known as esthesioneuroblastoma, is a malignant tumor of the nasal cavity believed to arise from the olfactory epithelium. ONB is rare, with a reported incidence of 0.4 per million (approximately 300 cases per year in the United States).
ONB most frequently arises in adults aged 20-60 years. Pediatric ONB is particularly rare, with only small case series or multi-institutional studies reported. However, a Surveillance Epidemiology and End Results (SEER) study of 47 pediatric patients with intranasal malignancy reported ONB as the most frequent pediatric intranasal malignancy (28%).
Due to the location of ONB in the sinonasal tract and anterior skull base, the presenting symptoms are often non-specific, including nasal obstruction and epistaxis, thus patients often present at advanced stages. Furthermore, ONB has a propensity for local invasion and relatively high rates of distant spread most commonly to the neck, lungs, and bones.
Clinical management in adults is generally surgical with adjuvant radiation.
The natural history of pediatric and adult ONB is incompletely understood, and treatment options for recurrent or metastatic ONB are limited. The planned natural history study as part of the NCI POB Rare Tumor Patient Engagement Network (RTPEN) will allow for comprehensive evaluation and recommendations to these patients while longitudinally collecting clinical, epidemiologic, and biological data.
Objective:
To characterize the natural history of ONB including clinical presentation, family history, patterns of disease progression, response to current treatment methods, disease recurrence, and overall survival
Eligibility:
Participants of age >= 3 years old with histologically documented ONB.
Design:
This protocol is intended to characterize the natural history of olfactory neuroblastoma.
Participants will undergo a comprehensive study entry evaluation including acquisition of imaging of tumor sites.
Medical histories will be documented, and participants followed throughout the course of their disease, with particular attention to patterns of disease recurrence and progression, response to therapies, duration of responses, and patient reported outcomes. Specimens will be obtained longitudinally, when feasible, and tumor growth rates will also be assessed throughout the course of the disease, when feasible.
Blood and tumor samples may be obtained at study entry and while on study.
- Conditions
- Olfactory Neuroblastoma, Esthesioneuroblastoma
- Keywords
- esthesioneuroblastoma, nasal cavity tumor, pediatric onb, adult onb, Natural History
- Medical subject headings
- Esthesioneuroblastoma, Olfactory
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Subjects with confirmed olfactory neuroblastoma. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize the natural history of ONB
To characterize the natural history of ONB including clinical presentation, family history, patterns of disease progression, response to current treatment methods, disease recurrence, and overall survival.
Measured: duration of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Feb 2021
- Study start2 Jun 2022
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Olfactory NeuroblastomaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 525 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Charalampos Floudas, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04755205
- Sponsor's protocol ID
- 210009
- Other IDs
- 21-C-0009
- Status last verified
- 13 Oct 2026
- First submitted
- 13 Feb 2021
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@@@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Natural History Study of Children and Adults With Olfactory Neuroblastoma
- ClinicalTrials.gov ID
- NCT04755205
- Acronym / sponsor ID
- 210009
- Conditions
- Olfactory Neuroblastoma, Esthesioneuroblastoma
- Keywords
- esthesioneuroblastoma, nasal cavity tumor, pediatric onb, adult onb, Natural History
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 525 participants (estimated)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 2 Jun 2022 (actual)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 16 Feb 2021 (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.
Background:
Olfactory neuroblastoma (ONB) is a rare cancer. It grows from tissue in the upper part of the nose cavity, related to the sense of smell and can affect a person s sense of smell. Researchers want to better understand the health problems of people with ONB. This may help them design better treatment and supportive care studies.
Objective:
To better understand ONB-the course of the disease, tumor characteristics, response to treatments, and management of the treatment.
Eligibility:
People ages 3 years and older who have ONB. They must enroll in NIH studies #19-C-0016 and #18-DC-0051.
Design:
Participants will be screened with a medical history and medical record review.
Participants do not have to visit NIH.
Participants will give a blood sample. They will complete surveys to assess their emotional and physical wellbeing and needs. Leftover tissue from biopsies and surgeries will be collected.
Participants will take smell tests. They will smell items and answer questions about them.
Participants may take taste tests. They will get plastic taste strips that they will move around their mouth to determine the taste.
Participants may have a physical exam. Their performance status may be assessed.
Participants may give blood, saliva, urine, and nasal secretion samples.
Participants may have computed tomography and/or magnetic resonance imaging scans.
Participants may have one or more tumor biopsies.
Participants will talk to the research team about the results of their medical record/tests evaluation. The team will recommend how to best manage and treat their disease.
Participants may give samples and complete surveys every 12 months. Their medical records will be reviewed every year. They will be monitored for the rest of their life.
Detailed description
Background:
Olfactory neuroblastoma (ONB), also known as esthesioneuroblastoma, is a malignant tumor of the nasal cavity believed to arise from the olfactory epithelium. ONB is rare, with a reported incidence of 0.4 per million (approximately 300 cases per year in the United States).
ONB most frequently arises in adults aged 20-60 years. Pediatric ONB is particularly rare, with only small case series or multi-institutional studies reported. However, a Surveillance Epidemiology and End Results (SEER) study of 47 pediatric patients with intranasal malignancy reported ONB as the most frequent pediatric intranasal malignancy (28%).
Due to the location of ONB in the sinonasal tract and anterior skull base, the presenting symptoms are often non-specific, including nasal obstruction and epistaxis, thus patients often present at advanced stages. Furthermore, ONB has a propensity for local invasion and relatively high rates of distant spread most commonly to the neck, lungs, and bones.
Clinical management in adults is generally surgical with adjuvant radiation.
The natural history of pediatric and adult ONB is incompletely understood, and treatment options for recurrent or metastatic ONB are limited. The planned natural history study as part of the NCI POB Rare Tumor Patient Engagement Network (RTPEN) will allow for comprehensive evaluation and recommendations to these patients while longitudinally collecting clinical, epidemiologic, and biological data.
Objective:
To characterize the natural history of ONB including clinical presentation, family history, patterns of disease progression, response to current treatment methods, disease recurrence, and overall survival
Eligibility:
Participants of age >= 3 years old with histologically documented ONB.
Design:
This protocol is intended to characterize the natural history of olfactory neuroblastoma.
Participants will undergo a comprehensive study entry evaluation including acquisition of imaging of tumor sites.
Medical histories will be documented, and participants followed throughout the course of their disease, with particular attention to patterns of disease recurrence and progression, response to therapies, duration of responses, and patient reported outcomes. Specimens will be obtained longitudinally, when feasible, and tumor growth rates will also be assessed throughout the course of the disease, when feasible.
Blood and tumor samples may be obtained at study entry and while on study.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | –Subjects with confirmed olfactory neuroblastoma. |
Who may qualify
- Ages
- 3 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with olfactory neuroblastoma.
Key inclusion criteria (3 listed)
- Subjects with histologically documented olfactory neuroblastoma.
- Age >= 3 years old.
- Ability of subject to understand and the willingness to sign a written consent document
Key exclusion criteria (1 listed)
- Pregnant women are excluded from this study because of more than minimal risk activities (imaging studies with contrast, biopsies) pertaining to enrollment.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Characterize the natural history of ONB
Time frame: duration of study
To characterize the natural history of ONB including clinical presentation, family history, patterns of disease progression, response to current treatment methods, disease recurrence, and overall survival.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. All IPD recorded in the medical record will be shared with intramural investigators upon request. @@@@@@@@@@@@In addition, all large scale genomic sequencing data will be shared with subscribers to dbGaP.
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A Phase 2 Study Of Zanzalintinib For Patients With Recurrent Or Metastatic Olfactory Neuroblastoma
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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.
Charalampos Floudas, M.D. principal investigator
National Cancer Institute (NCI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Olfactory Neuroblastoma and Esthesioneuroblastoma, run by National Cancer Institute (NCI) 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 3 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 16 Oct 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Marissa B Mallek, R.N. ((240) 760-7498, marissa.mallek@nih.gov), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterize the natural history of ONB (time frame: duration of study). 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 16 Oct 2026.