Exploring Outcomes and Risk in Patients With Rare Central Nervous System Tumors
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 326
- 试验地点
- 1
- 主要终点
- 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...
研究概览
简要总结
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.
详细描述
Background:
Rare cancers are defined as those with <40,000 cases per year. There are over 130 separate types of primary CNS tumors, all of which meet the definition of a rare cancer. However, some CNS cancers have incidences of less than 1,000 cases per year. Because of its relative rarity, limited reports of the presentation and clinical course have been completed. With the support of the CERN-Foundation, the Adult Ependymoma Outcomes Projects (AEO) was launched to address this lack of information for one type of rare CNS tumor- ependymoma. Using an online platform, participants and families from across the world have participated, providing information that has helped elucidate the presentation and ongoing health of individuals with this disease. To date, over 300 adults have participated with over 95% reporting interest in continuing and expanding their participation. Results from the AEO baseline survey and a follow-up survey (AEOII addressing socioeconomic impact of the disease) have been published. The most recent data from the AEO has been presented at the Society for Neuro-Oncology (SNO) annual meetings in 2015 and 2016.
An additional consequence of the relative rarity of these CNS tumors, studies to evaluate risk factors for the occurrence of these rare CNS tumors or predicting the clinical course of these rare CNS tumors are also limited. Rare CNS tumors like other cancers, occur when there are changes to genes that control the way cells grow and divide often as a result of exposure to other environmental risk factors. Therefore, exploring genetic changes in persons with rare CNS tumors will allow us to begin to understand what changes are associated specifically with these tumors. To date, these participants are often included as part of larger cohorts which include other types of brain tumors. We now understand that even among gliomas, the risk factors differ. Therefore, identifying the risk factors specifically associated with rare CNS tumors is critical for primary prevention and early detection. This knowledge would allow scientists and physicians to eventually screen for these changes or target the genes or the processes they control for treatment purposes.
Objectives:
The primary objectives of this study are to:
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •INCLUSION CRITERIA:
- •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.
排除标准
- •See inclusion criteria
研究组 & 干预措施
1/Patient with a rare CNS diagnosis
A diagnosis of rare CNS Tumors
结局指标
主要结局
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...
时间窗: completion of study
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.
次要结局
未报告次要终点