A Natural History Study of Children and Adults With Olfactory Neuroblastoma
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04755205 |
|
Recruitment Status :
Recruiting
First Posted : February 16, 2021
Last Update Posted : May 3, 2023
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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.
| Condition or disease |
|---|
| Olfactory Neuroblastoma Esthesioneuroblastoma |
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.
Subjects must also enroll on NIDCD Protocol 18-DC-0051.
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 Type : | Observational |
| Estimated Enrollment : | 500 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | A Natural History Study of Children and Adults With Olfactory Neuroblastoma |
| Actual Study Start Date : | June 2, 2022 |
| Estimated Primary Completion Date : | December 31, 2029 |
| Estimated Study Completion Date : | December 31, 2029 |
| Group/Cohort |
|---|
|
Cohort 1
Subjects with confirmed olfactory neuroblastoma.
|
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 3 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
- INCLUSION CRITERIA:
- Subjects must also enroll on NCI Protocol 18-DC-0051: "Biospecimen Procurement for NIDCD Clinical Protocols".
- Subjects with histologically documented olfactory neuroblastoma.
- Age >= 3 years old.
- Ability of subject to understand and the willingness to sign a written consent document
EXCLUSION CRITERIA:
Pregnant women are excluded from this study because of more than minimal risk activities (imaging studies with contrast, biopsies) pertaining to enrollment.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04755205
| Contact: Marissa B Mallek, R.N. | (240) 760-7498 | marissa.mallek@nih.gov | |
| Contact: Charalampos Floudas, M.D. | (240) 858-3032 | charalampos.floudas@nih.gov |
| United States, Maryland | |
| National Institutes of Health Clinical Center | Recruiting |
| Bethesda, Maryland, United States, 20892 | |
| Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937 | |
| Principal Investigator: | Charalampos Floudas, M.D. | National Cancer Institute (NCI) |
| Responsible Party: | National Cancer Institute (NCI) |
| ClinicalTrials.gov Identifier: | NCT04755205 |
| Other Study ID Numbers: |
210009 21-C-0009 |
| First Posted: | February 16, 2021 Key Record Dates |
| Last Update Posted: | May 3, 2023 |
| Last Verified: | January 23, 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | .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. |
| Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Informed Consent Form (ICF) |
| Time Frame: | Clinical data available during the study and indefinitely.@@@@@@Genomic data are available once genomic data are uploaded per protocol GDS plan for as long as database is active. |
| Access Criteria: | Clinical data will be made available via subscription to BTRIS and with the permission of the study PI. @@@@@@Genomic data are made available via dbGaP through requests to the data custodians. |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
|
esthesioneuroblastoma nasal cavity tumor pediatric onb adult onb Natural History |
|
Neuroblastoma Esthesioneuroblastoma, Olfactory Neuroectodermal Tumors, Primitive, Peripheral Neuroectodermal Tumors, Primitive Neoplasms, Neuroepithelial Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal |
Neoplasms by Histologic Type Neoplasms Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Olfactory Nerve Diseases Cranial Nerve Diseases Nervous System Diseases |