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临床试验/NCT02021734
NCT02021734已完成不适用

Genetics of Mendelian Diseases in Qatar

Weill Medical College of Cornell University1 个研究点 分布在 1 个国家目标入组 400 人开始时间: 2012年11月19日最近更新:
适应症

试验速览

阶段
不适用
状态
已完成
入组人数
400
试验地点
1
主要终点
Next generation sequencing

研究概览

简要总结

There are certain categories of diseases which are more prevalent in the Arab world due to increased rates of consanguinity in relatively isolated populations. The goal is to discover these mutations by using next-generation human genetics tools.

详细描述

There are certain categories of diseases which are more prevalent in the Arab world due to increased rates of consanguinity in relatively isolated populations. The goal is to discover these mutations by using next-generation human genetics tools. These include high-throughput sequencing and genotyping along with the necessary bioinformatics analyses that will lead to the discovery of the causes of most inherited diseases in the region.The secondary objective will be to build a comprehensive catalogue of genetic variation in the Arab world. This will include all detected mutations, not only the subset that are causing disease (from primary objective), but also known trait-altering mutations as well as general diversity on the DNA level among human populations of this region. This catalogue can become a widely useful resource for many projects down the road, as it relies on anonymizing individual samples and instead displaying data in aggregate as the cohorts of collected samples grow over the years.

The study will include all genetic disorders from all ethnic backgrounds but Mendelian disease for which a gene mutation has already been identified will be excluded.

Evidence of Mendelian Transmission determined by fulfilling one of the following criteria:

Multiple affected family members (at least first degree relative with disease) History of consanguinity Severe disease in newborn in the absence of family history Sydromic disease in single individuals Congenital abnormality affecting major organ system(s) Mendelianized extremes of common disease (eg sever familial diabetes/ obesity/ hypertension)

研究设计

研究类型
Observational
观察模型
Family Based
时间视角
Prospective

入排标准

性别
All
接受健康志愿者

入选标准

  • All included individuals must provide informed consent
  • All genetic disorders are included
  • All ethnic backgrounds are accepted
  • Disease must be genetic with no evident environmental cause
  • Evidence of Mendelian Transmission determined by fulfilling one of the following criteria:
  • Multiple affected family members (at least first degree relative with disease)
  • History of consanguinity
  • Severe disease in newborn in the absence of family history
  • Sydromic disease in single individuals
  • Congenital abnormality affecting major organ system(s)
  • Mendelianized extremes of common disease (eg sever familial diabetes/ obesity/ hypertension)

排除标准

  • Individuals who do no consent to be included
  • Mendelian disease for which a gene mutation has already been identified
  • Individuals for which a molecular diagnosis has already been established by alternative method
  • Disease for which an environmental factor is most likely the cause
  • Disease for which late age of onset rule out Mendelian transmission
  • Common diseases for which late age of onset rule out Mendelian transmission

结局指标

主要结局

Next generation sequencing

时间窗: 1 hour

Use next generation sequencing to detect novel disease causing mutations

次要结局

未报告次要终点

研究者

申办方类型
Other
责任方
Sponsor

研究点 (1)

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