International Rare Brain Tumor Registry
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- 1. Event-free survival, defined as time from start of treatment to an event (progression or recurrence of the disease, occurrence of a second malignant neoplasm, or death from any cause)
研究概览
简要总结
Rare brain tumors in children, adolescents, and young adults account for approximately 3-5% of all pediatric brain tumors. Due to the low incidence of some of these entities, there is a lack of standardized treatment approaches and thus poor outcomes. The recent incorporation of molecular information for diagnostic purposes has transformed the management of brain tumors. Nevertheless, some new molecularly defined entities are extremely rare and clinically poorly characterized.
We are planning to collect information on diagnosis, treatment, follow-up and additional information about your samples of tissues submitted at our hospital for better diagnosis purposes. The additional tests are optional. Further, we will also be sharing the images of the scans which will be done as part of diagnosis and treatment. This study is running in other countries with the same purpose. This will help us create a registry for future reference.
We aim to develop a bank where all such samples can be preserved as a repository biobank (A biobank is a collection of biological samples, like blood or tissue, that is carefully labeled with detailed information) for current and future research in children, adolescents, and young adults with rare brain tumors. However, the preservation and donation of specimens for research purposes is optional and one can still can continue participation in this study.
This is a retrospective and prospective observational study that seeks to collect matched tumor samples, clinical and radiological data to better understand the outcomes of patients with rare brain tumors. We are looking forward to take data on 30 patients retrospectively and 70 patients prospectively. The data will be shared with Children’s National Hospital, Washington DC, USA after obtaining necessary approvals It is the nodal centre maintaining the registry.
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 0.00 Year(s) 至 45.00 Year(s)（—）
- 性别
- All
入选标准
- •Retrospective Arm Patients with a known or suspected neoplasm that occurs in the pediatric, adolescent, or young adult populations are eligible for enrollment as follows: Diagnosis: 1- CNS Sarcomas 2- BCOR altered tumors 3- Astroblastoma/MNI-1 Altered tumors 4- Histologically ambiguous/Unclassifiable tumors 5- Other rare brain tumors (recently described, poorly characterized entities, etc.) Prospective Observational Arm Patients with a known or suspected neoplasm that occurs in the pediatric, adolescent, or young adult populations are eligible for enrollment as follows: Diagnosis:
- •CNS Sarcomas
- •BCOR altered tumors
- •Astroblastoma/MNI-1 Altered tumors
- •Histologically ambiguous/Unclassifiable tumors
- •Other rare brain tumors (recently described, poorly characterized entities, etc.).
排除标准
- •The patient has extra CNS tumors.
- •The patient is older than 46 years of age at diagnosis.
- •The patient or family is not willing to participate or does not sign informed consent (ONLY applicable for patients being considered for the Prospective Observational Arm).
结局指标
主要结局
1. Event-free survival, defined as time from start of treatment to an event (progression or recurrence of the disease, occurrence of a second malignant neoplasm, or death from any cause)
时间窗: 1. The study duration is 10 years, and subject duration is 1 year. every six months to probe patient status and collect radiological imaging and/or biospecimens if possible. Patient status at the date of last contact is also recorded. Relapse/Progression dates are recorded. | 2. Throughout the 10-year study duration. Cell lines may only be generated from fresh tissue of Prospective Arm study participants who provided informed consent. Samples collected will be stored indefinitely.
2. Successful establishment of reproducible cell lines and animal models derived from each cohort of rare brain tumors.
时间窗: 1. The study duration is 10 years, and subject duration is 1 year. every six months to probe patient status and collect radiological imaging and/or biospecimens if possible. Patient status at the date of last contact is also recorded. Relapse/Progression dates are recorded. | 2. Throughout the 10-year study duration. Cell lines may only be generated from fresh tissue of Prospective Arm study participants who provided informed consent. Samples collected will be stored indefinitely.
次要结局
- Characterize the clinicopathological characteristics of CNS sarcomas, BCOR altered tumors, Astroblastoma/MN1 altered tumors, & Unclassifiable brain tumors, & identify risk factors for survival & optimal therapeutic approaches.(Throughout the 10-year study duration. Demographics & disease characteristics are collected, including date of birth, date of diagnosis, age at diagnosis, date of death (if applicable), gender, race, ethnicity, clinical suspicion or confirmation of predisposition syndrome, institutional diagnosis, histological diagnosis, & neurological findings.)
- Utilize clinical, radiological, & biological data to develop a well-annotated biorepository biobank & therefore the infrastructure for current & future research.(Throughout the 10-year study duration. Biospecimen collection (tumor tissue, blood, CSF) is obtained when possible, & data points are collected at enrollment & biannually for prospective patients.)
- Identify & analyze conventional & advanced imaging findings (including PWI, fMRI, SWI, MRS, DTI) of rare pediatric brain tumors & correlate them with histopathology & genetic and/or molecular data.(Throughout the 10-year study duration. Radiological imaging (DICOM images) are collected at diagnosis (pre & postoperative) & for Prospective Arm patients, images are collected during biannual patient follow-ups.)
- Determine molecular characteristics of each cohort using somatic & germline SNV calling, methylation profiling, fusion calling, & gene expression profiling, & correlate molecular findings with clinical characteristics to identify risk factors & subgroup-specific therapeutic susceptibilities.(Throughout the 10-year study duration. Molecular analyses are performed on collected specimens, including DNA, RNA, & methylation analysis.)
- Analyze conventional & advanced imaging findings (including diffusion-weighted imaging) of each cohort & correlate them with histopathology & molecular data (radiogenomics)(Throughout the 10-year study duration. Radiological imaging (DICOM images) are collected at diagnosis (pre & postoperative) & for Prospective Arm patients, images are collected during biannual patient follow-ups.)
研究者
Dr Venkata Rama Mohan Gollamudi
Tata Memorial Hospital