Neoplasms

Nervous System Diseases

All Conditions

Rare Diseases

Esthesioneuroblastoma, Olfactory

Neuroblastoma

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Olfactory Nerve Diseases

Cranial Nerve Diseases

Olfactory Neuroblastoma

Neuroepithelioma

Ricardo Carrau, MD

The purpose of this study is to collect and organize data into a repository to gather relevant information for future research from patients diagnosed with a rare malignant sinonasal cancer called esthesioneuroblastoma (ENB) and are scheduled to undergo endonasal skull base surgery. Data being collected includes surgical data, demographics, disease presentation, MRI, CT, PET imaging, and post-operative assessments including pathology tests. After surgery, the investigators will collect chemotherapy and radiation data. Participants will also complete quality of life questionnaires at their follow-up visits.

Esthesioneuroblastoma: Outcomes After Treatment

Anterior Skull Base (ASB); Skull Base Inventory (SBI); Multidimensional Disease Specific Inventory MDS; ASB-Nasal 12; Sino-Nasal Outcome Test (SNOT-20)

Ricardo L. Carrau, MD

Subjects with confirmed olfactory neuroblastoma.

Marissa B Mallek, R.N.

Charalampos Floudas, M.D.

For more information at the NIH Clinical Center contact National Cancer Institute Referral Office

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.

...

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.

A Natural History Study of Children and Adults With Olfactory Neuroblastoma

.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.

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.

National Cancer Institute (NCI)

Skin and Connective Tissue Diseases

Mouth and Tooth Diseases

Carcinoma

Carcinoma, Squamous Cell

Melanoma

Salivary Gland Neoplasms

Carcinoma, Adenoid Cystic

Carcinoma, Mucoepidermoid

Mucoepidermoid Tumor

Adenocarcinoma

Neoplasms, Cystic, Mucinous, and Serous

Adenoid Cystic Carcinoma

Myoepithelial Carcinoma

Sinonasal Undifferentiated Carcinoma

Mucoepidermoid Carcinoma

Subjects treated at Massachusetts General Hospital with proton beam therapy

Intensity-modulated radiation therapy at institutions other than Massachusetts General Hospital

Proton radiation therapy

Intensity-modulated radiotherapy

Annie W Chan, MD

The purpose of this study is to test the hypothesis that 1)intensity-modulated radiotherapy (IMRT) or proton radiation therapy would result in improved local control rate and lowered toxicity compared to conventional radiotherapy, and 2) proton radiation therapy would result in equivalent or improved local control rate with similar or lower toxicity compared to IMRT, in the treatment of locally advanced sinonasal malignancy.

Data from retrospective studies suggest that IMRT or proton radiation therapy resulted in promising outcome in patients with sinonasal malignancy. To this date, no prospective study has been conducted to evaluate the outcome of sinonasal cancer treated with IMRT or proton radiation therapy. This Phase II trial is the first prospective study conducted to determine the treatment outcome and toxicity of IMRT or proton in the treatment of sinonasal cancer.

IMRT and proton radiation therapy are the two most established and most commonly employed advanced radiotherapy techniques for the treatment of sinonasal cancer. It is highly controversial whether one is superior to the other in terms of local control and toxicity outcome. It is also not clear if a subset of patients would benefit more from one treatment technology versus the other.

Due to the rarity and heterogeneity of sinonasal malignancies and the fact that proton beam is only available at a few centers in the United States, it is not feasible at present to do a Phase III study randomizing patients between IMRT and proton radiation therapy. In this study, a planned secondary analysis will be performed, comparing the treatment and toxicity outcome between IMRT and proton. The data on the IMRT and proton comparison from this trial will be used to design future multi-center prospective trials and to determine if randomized trial is necessary.

In this study, the treatment technique employed for an individual case will not be determined by the treating physician(s), but rather by the most advanced technology available at the treating institution for the treatment of the sinonasal cancer. At the Massachusetts General Hospital (MGH), proton beam therapy will be used for patients who meet the eligibility criteria. For institutions where protons are not available or institutions where the proton planning systems have not been optimized, IMRT exclusively will be used for the treatment of sinonasal cancer. Patient and tumor characteristics are expected to be comparable between IMRT- and proton- institutions

Subjects will receive daily proton radiation treatment as outpatients at the Francis H. Burr Proton Center at Massachusetts General Hospital. The subjects may also receive concurrent standard chemotherapy every week during their radiation therapy. This chemotherapy is considered standard treatment for their cancer and is not being done for research purposes.

In addition to daily radiation treatments, subjects will have the following tests every week: review of side effects; physical exam, including weight, height, neurological exam and vital signs; and blood tests, only for those subjects also receiving chemotherapy.

Subjects will be followed for 5 years after the completion of study treatment. The first follow-up visit will be 6-8 weeks after completion of study treatment. Additional follow-up visits will be performed every 3 months during the first 2 years following completion of radiation then every 6 month during years 3-5. At each follow-up visit, subjects will receive a physical exam, chest CT scan (at 1st follow-up visit then at least every 6 months), a CT or MRI tumor assessment (at 1st follow-up visit then at least every 6 months), quality-of-life questionnaire, hearing test, neuro-ophthalmology test, and neurocognitive test.

Intensity-Modulated or Proton Radiation Therapy for Sinonasal Malignancy

A Phase II Study of Intensity-Modulated or Proton Radiation Therapy for Locally Advanced Sinonasal Malignancy

To determine the local control rates with IMRT or proton radiation therapy at 2 years.

To determine the late visual-orbital late effects of IMRT or proton beam

To determine the regional control at 2 years after IMRT or proton

To determine the long-term survival at 5 years after IMRT or proton

To assess quality-of-life (QOL) outcomes after IMRT or proton

To determine the patterns of tumor relapse after IMRT or proton

To determine long-term local control after IMRT or proton

To determine long-term neurocognitive function after IMRT or proton

National Institutes of Health (NIH)

Massachusetts General Hospital

Assistant Professor

Gland and Hormone Related Diseases

Symptoms and General Pathology

Musculoskeletal Diseases

Diseases and Abnormalities at or Before Birth

Behaviors and Mental Disorders

Pituitary Diseases

Cysts

Chondrosarcoma

Meningioma

Pituitary Neoplasms

Craniopharyngioma

Adamantinoma

Chordoma

Encephalocele

Ameloblastoma

Central Nervous System Cysts

Hypothalamic Diseases

Brain Diseases

Central Nervous System Diseases

Endocrine System Diseases

Neoplasms, Vascular Tissue

Meningeal Neoplasms

Central Nervous System Neoplasms

Nervous System Neoplasms

Neoplasms, Connective Tissue

Neoplasms, Connective and Soft Tissue

Sarcoma

Endocrine Gland Neoplasms

Hypothalamic Neoplasms

Supratentorial Neoplasms

Brain Neoplasms

Bone Neoplasms

Bone Diseases

Congenital Abnormalities

Nervous System Malformations

Spinal Dysraphism

Neural Tube Defects

Hernia

Pathological Conditions, Anatomical

Quality of Life

Soft Tissue Sarcoma

Megan Frost, BS

Kyle Wu, MD

Daniel Prevedello, MD

This is a prospective longitudinal study to access postoperative 2-year quality of life in patients who undergo endonasal endoscopic approach surgeries of the skull base.

This is a prospective single-institution longitudinal study to access quality of life in patients who undergo endonasal endoscopic approach surgeries of the skull base. Patients will be followed before surgery and up to twenty-four months post-surgery. The primary goal of the study is to assess the alterations to patient quality of life following endoscopic endonasal surgery using modern sinonasal reconstruction techniques. Secondary goals are to better understand the time course of normal healing and patient/procedural risk factors associated with poorer quality of life outcomes.

Long-Term Longitudinal QoL in Patients Undergoing EEA

A Prospective Longitudinal Quality of Life Study in Patients Undergoing Endoscopic Endonasal Skull Base Surgery

There will be no sharing of individual participant data with other researchers. Data will be de-identified once analysis is complete and destroyed once manuscripts are written.

This study aims to better understand the long-term overall quality of life of patients undergoing endoscopic endonasal surgery using modern sinonasal reconstruction techniques. The PROMIS-29 metric, an overall quality of life metric, will be used to better understand quality of life for patients undergoing this procedure.

This study aims to better understand the overall nose function in patients undergoing endoscopic endonasal surgery using modern sinonasal techniques. The ASK Nasal-12 metric is designed to evaluate nose overall nose function such as post-nasal drip, ability to smell, and pain.

This study aims to better understand the number of long-term risk factors for patients undergoing endoscopic endonasal surgeries using modern sinonasal reconstruction techniques. This will be done using the PROMIS-29 metric along with patient data.

This study aims to better understand the number of long-term comorbidities for patients undergoing endonasal endoscopic surgeries using modern sinonasal reconstruction techniques. This will be done using the ASK-Nasal 12 metric along with patient data.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Esthesioneuroblastoma: Outcomes After Treatment	NCT02580136	Esthesioneurobl...		10 Years - 80 Years	Ohio State University
A Natural History Study of Children and Adults With Olfactory Neuroblastoma	NCT04755205	Olfactory Neuro... Esthesioneurobl...		3 Years - 120 Years	National Institutes of Health Clinical Center (CC)
Esthesioneuroblastoma: Outcomes After Treatment	NCT02580136	Esthesioneurobl...		10 Years - 80 Years	Ohio State University
Intensity-Modulated or Proton Radiation Therapy for Sinonasal Malignancy	NCT01586767	Adenoid Cystic ... Squamous Cell C... Sinonasal Carci... Sinonasal Undif... Mucoepidermoid ... Schneiderian Ca... Myoepithelial C... Esthesioneurobl... Melanoma	Proton radiatio... Intensity-modul...	18 Years -	Massachusetts General Hospital
Long-Term Longitudinal QoL in Patients Undergoing EEA	NCT04087902	Pituitary Tumor Meningioma Rathke Cleft Cy... Chordoma Chondrosarcoma Craniopharyngio... Encephalocele Esthesioneurobl...		18 Years -	Ohio State University

Spots Global Cancer Trial Database for esthesioneuroblastoma