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Safety assessment of gene therapy in major genetic related diseases
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注册号:

Registration number:

ChiCTR1800018955 

最近更新日期:

Date of Last Refreshed on:

2018-11-24 

注册时间:

Date of Registration:

2018-10-18 

注册号状态:

补注册  

Registration Status:

Retrospective registration  

注册题目:

重大遗传疾病基因治疗的安全性评估 

Public title:

Safety assessment of gene therapy in major genetic related diseases 

注册题目简写:

 

English Acronym:

 

研究课题的正式科学名称:

人类废弃胚胎,小鼠和猴子胚胎基因编辑安全性评估 

Scientific title:

Evaluating the safety of genome editing in abandoned human embryos and in monkey and mouse embryos 

研究课题代号(代码):

Study subject ID:

 

在二级注册机构或其它机构的注册号:

The registration number of the Partner Registry or other register:

 

申请注册联系人:

覃金洲 

研究负责人:

贺建奎 

Applicant:

QIN JINZHOU 

Study leader:

He Jiankui 

申请注册联系人电话:

Applicant telephone:

+86 13246755209 

研究负责人电话:

Study leader's telephone:

+86 18688955436 

申请注册联系人传真 :

Applicant Fax:

 

研究负责人传真:

Study leader's fax:

 

申请注册联系人电子邮件:

Applicant E-mail:

qinjinzhou851018@163.com 

研究负责人电子邮件:

Study leader's E-mail:

hejk@sustc.edu.cn 

申请单位网址(自愿提供):

Applicant website(voluntary supply):

 

研究负责人网址(自愿提供):

Study leader's website(voluntary supply):

 

申请注册联系人通讯地址:

中国深圳罗湖区春风路47号 

研究负责人通讯地址:

中国深圳南山区学苑大道1088号 

Applicant address:

47 Chunfeng Road, Luohu, Shenzhen, Guangdong, China 

Study leader's address:

1088 Xueyuan Road, Shenzhen, Guangdong, China 

申请注册联系人邮政编码:

Applicant postcode:

 

研究负责人邮政编码:

Study leader's postcode:

 

申请人所在单位:

深圳大学第三附属医院 

Applicant's institution:

Third Affiliated Hospital of Shenzhen University 

是否获伦理委员会批准:

是 

Approved by ethic committee:

Yes 

伦理委员会批件文号:

Approved No. of ethic committee:

2017015001 

伦理委员会批件附件:

Approved file of Ethical Committee:

查看附件View

批准本研究的伦理委员会名称:

深圳大学第三附属医院医学伦理委员会 

Name of the ethic committee:

Medical Ethics Committee of the Third Affiliated Hospital of Shenzhen University 

伦理委员会批准日期:

Date of approved by ethic committee:

 

伦理委员会联系人:

张秀明 

Contact Name of the ethic committee:

Zhang Xiuming 

伦理委员会联系地址:

中国深圳罗湖区春风路47号 

Contact Address of the ethic committee:

47 Chunfeng Road, Luohu, Shenzhen, Guangdong, China 

伦理委员会联系人电话:

Contact phone of the ethic committee:

 

伦理委员会联系人邮箱:

Contact email of the ethic committee:

 

研究实施负责(组长)单位:

南方科技大学 

Primary sponsor:

South University of Science and Technology of China 

研究实施负责(组长)单位地址:

中国深圳南山区学苑大道1088号 

Primary sponsor's address:

1088 Xueyuan Road, Shenzhen, Guangdong, China 

试验主办单位(项目批准或申办者):

Secondary sponsor:

国家:

中国

省(直辖市):

广东

市(区县):

深圳

Country:

China

Province:

Guangdong

City:

Shenzhen

单位(医院):

深圳大学第三附属医院

具体地址:

中国深圳,罗湖区春风路47号

Institution
hospital:

Third Affiliated Hospital of Shenzhen University

Address:

47 Chunfeng Road, Luohu, Shenzhen, Guangdong, China

国家:

中国

省(直辖市):

广东

市(区县):

深圳

Country:

China

Province:

Guangdong

City:

Shenzhen

单位(医院):

南方科技大学

具体地址:

中国深圳,南山区学苑大道1088号

Institution
hospital:

South University of Science and Technology of China

Address:

1088 Xueyuan Road, Shenzhen, Guangdong, China

国家:

中国

省(直辖市):

湖南

市(区县):

Country:

China

Province:

Hu'nan

City:

单位(医院):

中信湘雅生殖与遗传专科医院

具体地址:

中国湖南省长沙市湘雅路84号

Institution
hospital:

CITIC-XIANGYA Genetics & Reproduction Hospital

Address:

84 Xiangya Road, Changsha, Hu'nan, China

国家:

中国

省(直辖市):

河南

市(区县):

Country:

China

Province:

He'nan

City:

单位(医院):

郑州大学第三附属医院

具体地址:

中国河南省郑州市二七区康复前街7号

Institution
hospital:

Third Affiliated Hospital of Zhenzhou University

Address:

7 Kangfu Street, Erqi District, Zhengzhou, He'nan

国家:

中国

省(直辖市):

云南

市(区县):

Country:

China

Province:

Yunan

City:

单位(医院):

昆明理工大学

具体地址:

中国云南省昆明市一二一大街文昌路68号

Institution
hospital:

Kunming University of Science and Technology

Address:

68 Wenchang Road, Yieryi Street, Kunming, Yunnan, China

经费或物资来源:

深圳市科技创新自由探索项目 

Source(s) of funding:

Shenzhen Science and Technology Innovation Free Exploration Project 

研究疾病:

重大遗传疾病 

Target disease:

Major genetic disease 

研究疾病代码:

 

Target disease code:

 

研究类型:

干预性研究 

Study type:

Interventional study 

研究所处阶段:

基础科学研究 

Study phase:

Basic Science 

研究目的:

基因疗法就是通过基因工程技术手段,来取代或矫正患者或实验动物缺陷基因,以达到根治遗传疾病的目的。2017年全世界首款基因疗法在美国FDA获批,同时首个人体内基因编辑临床试验开始。本研究拟采用当今最热门CRISPR-Cas9基因编辑技术,结合其衍射的单剪辑编辑技术,对人类辅助生殖技术中经过患者知情同意废弃胚胎(主要为3PN胚胎)进行探究性试验,目前我们基于动物实验(小鼠和猴子胚胎实验结果),选定CCR5和PCSK9作为研究目标基因。通过该研究,精确修复遗传突变导致的疾病,或调解致病基因表达,预防相关疾病。同时,可根据对样本的深度测序分析,关联相关基因,分析不孕不育发病机理,改善不孕不育患者妊娠结局。 

Objectives of Study:

Gene therapy is to replace or correct the defective genes of patients by genetic engineering technology, so as to achieve the goal of eradicating hereditary diseases. In 2017, the first gene therapy was approved in the United States, and the first human genome editing clinical trial started. In this study, we will use the most popular CRISPR/Cas9 gene editing technology and its derivative single base mutation technology to carry out exploratory experiments on the abandoned human embryo samples (mainly 3PN samples) from the assisted reproduction process. The donors of the human embryos are consent informed. At present, we have selected the CCR5 and PCSK9 genes as the research targets. Through this study, point mutations in diseases are accurately repaired to prevent the disease, or to regulate the expression of the pathogenic gene, so as to cure the related diseases. At the same time, the related samples will be sequenced in depth, and the related genes can be analyzed to reveal the mechanism of infertility to improve the pregnancy outcome of infertility. 

药物成份或治疗方案详述:

 

Description for medicine or protocol of treatment in detail:

 

研究设计:

随机平行对照试验 

Study design:

Randomized parallel controlled trial 

纳入标准:

①以女方输卵管堵塞因素导致的不孕为病因来源;②年龄23~43岁;③3个月内未接受影响内分泌功能的药物治疗;④夫妻均自愿参与;⑤使用长方案促排卵方案。 

Inclusion criteria

1. Infertility caused by female fallopian tube obstruction; 2. aged 23 to 43 years old; 3. no drug treatment affecting endocrine function within 3 months; 4. the couple to participate voluntarily; 5. long-term ovulation promotion program was used; 

排除标准:

①患有严重生理疾病或精神疾病者(包括恶性肿瘤患者);②经项目组成员解释后,拒绝参与者;③患有内分泌相关疾病,影响激素水平的准确测定的患者。 

Exclusion criteria:

1. Patients with severe physical or mental illness (including malignant tumors); 2. denied to participate after explanation by the project team members; 3. patients with endocrine-related diseases that affect the accurate determination of hormone levels.  

研究实施时间:

Study execute time:

From2017-04-01To 2020-04-01 

干预措施:

Interventions:

组别:

A

样本量:

200

Group:

A

Sample size:

干预措施:

Cas9 RNA

干预措施代码:

Intervention:

Cas9 RNA

Intervention code:

组别:

B

样本量:

200

Group:

B

Sample size:

干预措施:

Cas9 蛋白

干预措施代码:

Intervention:

Cas9 Protein

Intervention code:

研究实施地点:

Countries of recruitment and research settings:

国家:

中国 

省(直辖市):

广东 

市(区县):

深圳 

Country:

China 

Province:

Guangdong 

City:

Shenzhen 

单位(医院):

深圳大学第三附属医院 

单位级别:

三甲医院 

Institution
hospital:

Third Affiliated Hospital of Shenzhen University  

Level of the institution:

Tertiary A Hospital 

测量指标:

Outcomes:

指标中文名:

切割效率

指标类型:

主要指标 

Outcome:

On target rate

Type:

Primary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

脱靶情况

指标类型:

主要指标 

Outcome:

off target

Type:

Primary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

采集人体标本:

Collecting sample(s)
from participants:

标本中文名:

废弃胚胎

组织:

Sample Name:

Abandoned embryo

Tissue:

人体标本去向

使用后销毁 

说明

Fate of sample:

Destruction after use 

Note:

征募研究对象情况:

Recruiting status:

正在进行

Recruiting

年龄范围:

Participant age:

最小 Min age years
最大 Max age years

性别:

男女均可

Gender:

Both

随机方法(请说明由何人用什么方法产生随机序列):

按照样本顺序标号,随机数表法提取胚胎,分组使用

Randomization Procedure (please state who generates the random number sequence and by what method):

According to the sample order labels, the table of random numbers method is used to extract the embryos for use in groups.

盲法:

N/A

Blinding:

N/A

试验完成后的统计结果(上传文件):

点击下载

Calculated Results ater
the Study Completed(upload file):

download

原始数据公开时间:

The time of sharing IPD:

Within six months after the trial complete

共享原始数据的方式(说明:请填入公开原始数据日期和方式,如采用网络平台,需填该网络平台名称和网址):

暂不公开

The way of sharing IPD”(include metadata and protocol, If use web-based public database, please provide the url):

Temporarily not public

数据采集和管理(说明:数据采集和管理由两部分组成,一为病例记录表(Case Record Form, CRF),二为电子采集和管理系统(Electronic Data Capture, EDC),如ResMan即为一种基于互联网的EDC:

暂不公开

Data collection and Management (A standard data collection and management system include a CRF and an electronic data capture:

暂不公开

数据管理委员会:

Data Managemen Committee:

暂未确定/Not yet

注册人:

Name of Registration:

 2018-10-18
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