Molecular Targets for the Treatment of Histiocytosis
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 1,800
- 试验地点
- 3
- 主要终点
- Molecular alterations in histiocytosis
研究概览
简要总结
The primary objective: to develop technical and operating procedures for detection mutations of histiocytosis during clinical practice of no-specialized molecular platforms, for diagnosis and follow-up of the disease.
The secondary objectives: to describe therapeutic target mutations in histiocytosis patients, and to develop the cellular tests to evaluate in vitro the sensibility of these mutations drive to inhibitors.
详细描述
The study is ancillary of the French "Gene Histio" cohort (patients already included) and "HISTIO target 2020" cohort (patients recruited since may 2020).
The data base of the French registry of histiocytoses is held by Dr J. Donadieu, and localized in Trousseau hospital (APHP) 75012 Paris, France, where server and backup are kept.
5 teams contributes to the study with different role: Team 1 is responsible for histology diagnosis, selection of histiocyte- rich areas, extraction of nucleic acids and detection of the BRAF p.V600E mutations. This group is also responsible of the tissue and nucleic acids biobank.
Team 2 is responsible for the collection of clinical data and tissue and blood samples of the children. This group is also responsible of the clinical data base.
Team 3 is responsible for the collection of clinical data and tissue and blood samples of the adult patients.
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
- •Histologically proven histiocytosis;
- •Informed consent form signed by patients (or parents/legal tutors of children) to participate"Gene Histio" or "HISTIO target 2020".
排除标准
- •Patient refusal.
结局指标
主要结局
Molecular alterations in histiocytosis
时间窗: throughout of the study, an average of 3 years
Characterize the molecular alterations of nucleic acids in histiocytosis. Assess the frequency and type of drugable mutations in the different sub-types and localizations of histiocytosis Molecular alterations will be assessed by next analysis of DNA or RNA extracted from histiocytosis tissue or blood samples.
次要结局
未报告次要终点