Childhood Cancer Predisposition Study (CCPS)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,050
- 试验地点
- 7
- 主要终点
- Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies.
研究概览
简要总结
The Childhood Cancer Predisposition Study (CCPS) is a multi-center, longitudinal, observational study that will collect clinical and biological data and specimens from children with a cancer predisposition syndromes (CPS) and their relatives.
The central hypothesis is that studying individuals at high risk for childhood cancer creates a unique opportunity for improving the understanding of carcinogenesis, tumor surveillance, early detection, and cancer prevention, which will collectively contribute to improving care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
详细描述
The CCPS is designed as a multi-center, longitudinal, observational study that will collect clinical and biological data and specimens from children with a CPS and their relatives. The investigators plan to:
- Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies;
- Define the natural history of disease in children with CPS; and
- Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies.
The study will enroll approximately 350 Children and 700 Relatives per year. The investigators plan to collect demographic and diagnostic data at enrollment. Longitudinal follow-up will be performed at least annually.
The CCPS includes the establishment of a biorepository, with a hub and spoke structure, with a central repository at Emory University/Children's Healthcare of Atlanta for prospective collection of some tissues, linked to local biorepositories at participating institutions. Information about inventory of tumor specimens already banked locally will be available in the database, along with reference to existing genomic studies of the tumor, such that investigators may identify and request such tissue or data for specific studies, subject to approval of the CCPS Scientific Committee.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Primary Subjects must meet all of the below criteria to be eligible for enrollment:
- •Be less than 21 years of age at the time of enrollment
- •Have a diagnosis of a specific CPS, whether they have had cancer or not
- •Based on clinical laboratory testing demonstrating a Pathogenic or Likely Pathogenic germline variant and/or
- •Based on well-established clinical diagnostic criteria and/or
- •Based on high clinical suspicion of a specific CPS with clinical laboratory testing demonstrating a variant of uncertain significance (VUS)
- •Affected Parents must meet all of the following criteria to be eligible for enrollment:
- •Be the biologic parent of a Primary Subject and
- •Carry a diagnosis (or obligate diagnosis) of the familial CPS
- •Adult Affected Siblings must meet all of the following criteria to be eligible for enrollment:
- •Be the biologic sibling of a Primary Subject and
- •Carry a diagnosis (or obligate diagnosis) of the familial CPS
- •Unaffected Parents and Siblings must meet all of the following criteria to be eligible for enrollment
- •Be the biologic parent or sibling of a Primary Subject and
- •Not carry a diagnosis (or obligate diagnosis) of the familial CPS
- •Affected Family Members must meet all of the following criteria to be eligible for enrollment:
- •1. Carry a diagnosis of (or obligate diagnosis of) the familial CPS. Documentation is requested but not required.
- •More than one child from a Primary Family Unit may be a Primary Subject. An Unaffected Sibling may be reclassified as a Primary Subject if diagnosed with a CPS during childhood.
排除标准
- •Individuals with a strong personal or family history of cancer without a genetic or clinical diagnosis of a specific CPS are not eligible for enrollment.
结局指标
主要结局
Establish and maintain a framework for recruitment, participation, and surveillance of children with cancer predisposition syndromes (CPS) in clinical and translational research studies.
时间窗: Up to 10 years
This multicenter registry and biorepository will be developed with the purpose of studying individuals at high risk for childhood cancer to improve care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
Define the natural history of disease in children with CPS.
时间窗: Up to 10 years
To define the natural history of disease in children with CPS.
Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies.
时间窗: Up to 10 years
Evaluate the clinical impact and effectiveness of standard and emerging tumor surveillance strategies to improve care and outcomes for pediatric patients with cancer and those with cancer predisposition syndromes (CPS).
次要结局
未报告次要终点
研究者
Christopher Porter
Associate Professor
Emory University