Rare CNS Tumors Outcomes &Risk

NCT03251989 | Completed

• 2 other identifiers: 99991714117-C-N141
• Study Type: observational
• Target: 326
• Locations: 1 country
• Sites: 1

Brief Summary

Background: Primary tumors of the brain and spine are those that start in the brain or spine. These tumors are rare, accounting for \<2% of all cancers diagnosed in the United States. Some of these tumors occur in less than 2,000 people per year. Researchers want to study a large group of people with this kind of tumor. They want to learn more about the tumors, including the risk factors related to how they develop in adults. Objective: To collect health and gene data to learn about what changes are associated with a rare CNS Tumors, to eventually screen for these changes or target the genes in treatment. Eligibility: Adult participants \>= 18 years of age who self- identify as being diagnosed with one of 12 rare CNS tumors, including: Atypical teratoid rhabdoid tumor (ATRT); Brainstem and midline gliomas; Choroid plexus tumors; Ependymoma; High grade meningioma; Gliomatosis cerebri; Medulloblastoma; Oligodendroglioma / Anaplastic oligodendroglioma; Pineal region tumors; Pleomorphic xanthroastrocytoma / Anaplastic pleomorphic xanthroastrocytoma; PNET (Supratentorial embryonal tumor); Primary CNS sarcoma / Secondary CNS sarcoma (Gliosarcoma). Design: Participants will be invited to participate through an ad on the CERN Foundation website (ependymoma), information on the Neuro-Oncology Branch website and other identified advocacy and social media sites and direct mailer to those who have already participated in the EO projects. (Registered Trademark)

• Interested participants will complete an enrollment form that will be sent to the study coordinator.
• The coordinator will then send the participant a consent form and schedule a time for phone consent.
• Participants will complete the Rare CNS tumors Outcomes Survey and once completed, the Rare CNS tumors Risk survey. (Registered Trademark)
• The questions on the Outcomes Survey will include treatment history, symptoms social and clinical information and it should take about 25-35 minutes. The Risk survey will cover their demographic information, personal medical history, family medical history and environmental exposures. This should take about 52 minutes.
• Participants who have physical problems can have help with the surveys and forms.
• Once the surveys are completed, participants will be mailed a kit to collect saliva for germline DNA. Participants will ship the sample to the study team in a prepaid envelope
• If the sample is not sufficient, participants will be contacted to give provide an additional sample.

Conditions

High Grade Meningioma; Ependymoma; Medulloblastoma; PNET; Primary CNS Sarcoma

Keywords

Brain Tumor; Central Nervous System Tumors; Choroid Plexus Tumors; Gliomas; Atypical Teratoid Rhabdoid Tumor; Natural History

Outcome Measures

Primary Outcomes (1)

• Relationship between health status and disease and treatment characteristics as well as clinical and demographic risk factors as self reported by adult participants with rare CNS tumors; and the relationship of genomic susceptibility of the popu...

  Obtain self-reported data on treatment, symptoms, functional status, and quality of life for adult participants with rare CNS tumors To evaluate the relationship between health status and disease and treatment characteristics. To evaluate self-reported clinical and demographic risk factors in adult participants in the rare CNS tumors participant population. To explore genomic susceptibility in participants with rare CNS tumors.

  completion of study

Study Arms (1)

1/Patient with a rare CNS diagnosis

A diagnosis of rare CNS Tumors

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Participants will be solicited through the Collaborative Ependymoma Research Network (CERN), information on the Neuro-Oncology Branch website and other advocacy organization websites, mailing list and social media sites.@@@

You may qualify if:

• Participants with rare CNS tumors who meet the following criteria will be invited to participate in the study:
• A diagnosis of rare CNS tumors, (Atypical teratoid rhabdoid tumor (ATRT); Brainstem and midline gliomas; Choroid plexus tumors; Ependymoma; High grade meningioma; Gliomatosis cerebri; Medulloblastoma; Oligodendroglioma / Anaplastic oligodendroglioma; Pineal region tumors; Pleomorphic xanthroastrocytoma / Anaplastic pleomorphic xanthroastrocytoma; PNET (Supratentorial embryonal tumor); Primary CNS sarcoma / Secondary CNS sarcoma (Gliosarcoma) or as reported by the participant:
• Participants who are currently \>= 18 years of age with the initial rare CNS tumor diagnosis occurring at any point in their lifetime are eligible.
• Ability to speak, write, and read English, as questionnaires available in English language only.
• Ability of participant to understand and the willingness to sign a written informed consent document.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Cancer Institute (NCI)/ Neuro-Oncology Branch

Bethesda, Maryland, 20892, United States

Location

Related Publications (4)

• Armstrong TS, Vera-Bolanos E, Gilbert MR. Clinical course of adult patients with ependymoma: results of the Adult Ependymoma Outcomes Project. Cancer. 2011 Nov 15;117(22):5133-41. doi: 10.1002/cncr.26181. Epub 2011 Apr 28.

  PMID: 21538344 BACKGROUND

• Walbert T, Mendoza TR, Vera-Bolanos E, Acquaye A, Gilbert MR, Armstrong TS. Symptoms and socio-economic impact of ependymoma on adult patients: results of the Adult Ependymoma Outcomes Project 2. J Neurooncol. 2015 Jan;121(2):341-8. doi: 10.1007/s11060-014-1638-4. Epub 2014 Oct 31.

  PMID: 25359395 BACKGROUND

• Acquaye AA, Vera E, Gilbert MR, Armstrong TS. Clinical presentation and outcomes for adult ependymoma patients. Cancer. 2017 Feb 1;123(3):494-501. doi: 10.1002/cncr.30355. Epub 2016 Sep 28.

  PMID: 27679985 BACKGROUND

• McIver BA, Davis TS, Reinhart K, Vera E, Acquaye-Mallory A, Choi A, Kunst T, Johnson M, Grajkowska E, Miller H, Reyes J, Gilbert MR, Armstrong TS, Wright ML. Evaluating Clinical and Sociodemographic Risk for Symptom Burden Associated Interference With Daily Functioning in the Primary Brain Tumor Patient Population. Cancer Med. 2025 Mar;14(5):e70682. doi: 10.1002/cam4.70682.

  PMID: 40052556 DERIVED

MeSH Terms

Conditions

Ependymoma; Medulloblastoma; Neuroectodermal Tumors, Primitive; Brain Neoplasms; Central Nervous System Neoplasms; Choroid Plexus Neoplasms; Glioma; Rhabdoid Tumor

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Ventricle Neoplasms; Neoplasms, Complex and Mixed

Study Officials

• Tito R Mendoza, Ph.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 15, 2017
• First Posted: August 16, 2017
• Study Start: August 21, 2017
• Primary Completion: May 6, 2025
• Study Completion: May 6, 2025
• Last Updated: March 24, 2026

Record last verified: 2025-12-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, 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. @@@@@@All collected IPD will be available after primary analysis have been published.
• 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.

Locations

US (1)