Today is 2018-12-14

Been withdrawn with the reason of the original applicants cannot provide the individual participants data for reviewing Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos
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注册号:

Registration number:

ChiCTR1800019378 

最近更新日期:

Date of Last Refreshed on:

2018-11-30 

注册时间:

Date of Registration:

2018-11-08 

注册号状态:

补注册  

Registration Status:

Retrospective registration  

注册题目:

因不能提供原始数据供审核,已驳回补注册申请 HIV免疫基因CCR5胚胎基因编辑安全性和有效性评估 

Public title:

Been withdrawn with the reason of the original applicants cannot provide the individual participants data for reviewing Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos 

注册题目简写:

 

English Acronym:

 

研究课题的正式科学名称:

基因编辑人类胚胎CCR5基因安全性和有效性评估 

Scientific title:

Evaluation of the safety and efficacy of gene editing with human embryo CCR5 gene 

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:

中国深圳南山区学苑大道1088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:

1088 Xueyuan Road, Nanshan, Shenzhen, Guangdong, China 

Study leader's address:

1088 Xueyuan Road, Nanshan, 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:

南方科技大学 

Applicant's institution:

Southern University of Science and Technology 

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:

20170307 

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:

深圳和美妇儿科医院伦理委员会 

Name of the ethic committee:

Medical Ethics Committee of Shenzhen HOME Women's and Children's Hospital 

伦理委员会批准日期:

Date of approved by ethic committee:

2017-03-07 

伦理委员会联系人:

黄华峰 

Contact Name of the ethic committee:

Huang Huafeng 

伦理委员会联系地址:

中国深圳市南山区深南大道12018号 

Contact Address of the ethic committee:

12018 Shenlan Road, Nanshan, 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, Nanshan, 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):

Country:

China

Province:

Guangdong

City:

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, Nanshan, Shenzhen, Guangdong, China

国家:

中国

省(直辖市):

广东

市(区县):

Country:

China

Province:

Guangdong

City:

单位(医院):

深圳和美妇儿医院

具体地址:

中国深圳,深圳市南山区深南大道12018号

Institution
hospital:

Shenzhen HarMoniCare Women & Children’s Hospital

Address:

12018 Shenlan Road, Nanshan, Shenzhen, 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:

HIV 

Target disease:

HIV 

研究疾病代码:

 

Target disease code:

 

研究类型:

相关因素研究 

Study type:

Relative factors research 

研究所处阶段:

其它 

Study phase:

Other 

研究目的:

HIV导致的AIDS是的当今世界威胁全人类的一大医学难题,影响着全人类的生命安全和身体健康。迄今为止,没有有效的药物或临床技术手段可完全治愈AIDS。所幸全世界各国政府和科学家投入大量精力做HIV预防和感染后干预,但是距离WHO关于2020年HIV防治目标任然很远,距离消除HIV任重道远。迄今为止唯一一例全世界被公认完全治愈的HIV感染者是“柏林病人”,当时患者出现白血病,在做骨髓干细胞移植前检查出为HIV阳性感染者,德国医生采用了一个西欧人群中罕见可以抵御HIV-1的基因突变-具有CCR5突变的骨髓配型的创造性的治疗了该患者的白血病,同时到今天为止“柏林病人”的体内已检测不到HIV病毒,为消灭HIV创造了新的医疗模式。本试验在细胞系、动物实验和人类废弃胚胎的基础上,招募罹患不孕不症的HIV阳性患者,通过充分的知情同意告知志愿者风险及获益,通过一对一面谈,签署知情同意书;同时提交合作医院伦理委员会讨论并通过实验设计。通过CCR5基因编辑人类胚胎,通过完善的试验体系,获得避免HIV健康小孩,为未来在人类早期胚胎彻底消除重大遗传疾病提供了新见解。 

Objectives of Study:

HIV-induced AIDS is a major medical problem that threatens all human beings in today's world, affecting the safety and health of all human beings. To date, there is no effective drug or clinical technique to completely cure AIDS. Fortunately, governments and scientists around the world have invested a lot of energy in HIV prevention and post-infection interventions. However, we are far from achieving the WHO's 2020 HIV prevention goals and have a long way to go to eliminate HIV. The only HIV-infected person who has been recognized as completely cured in the world is the Berlin patient”. At that time, the patient developed leukemia and was diagnosed as HIV-positive before the bone marrow stem cell transplant. The German doctor used a bone marrow matching to creatively treats leukemia in this patient with a rare CCR5 genetic mutation existing in Western European population resistant to HIV-1. To date, "Berlin patient" has not detected with HIV in the body, creating a new medical model for HIV elimination. The current clinical trial is based on preclinical research of cell lines, animal models and human waste embryos. It recruits HIV-positive patients with infertility and informs the volunteers of the risks and benefits through sufficient informed consenting. The informed consent form is signed through one-on-one discussion. The study design was submitted to the ethics committee of the hospital for discussion and approval. Through the CCR5 gene editing of the human embryo in a comprehensive test system, we set to obtain healthy children to avoid HIV providing new insights for the future elimination of major genetic diseases in early human embryos. 

药物成份或治疗方案详述:

 

Description for medicine or protocol of treatment in detail:

 

研究设计:

连续病例 

Study design:

Case series 

纳入标准:

生活在中华人民共和国的已婚夫妇艾滋病毒血清阳性(女性阴性,男性阳性) 男女22–38岁 男性临床稳定,检测不到病毒载量血症<75拷贝/ mL;筛选时CD4计数> 250;至少在过去12个月内有连续抗逆转录病毒疗法的历史 临床证实满足试管婴儿(IVF)医学指针 受试者双方的充分知情同意,理解本试验目的、风险和利益 受试者双方都愿意承诺在卵子收集前和出生后一个月内使用预防性避孕或保持禁欲至少两个月 

Inclusion criteria

1. Married couple living in the People's Republic of China with HIV seropositivity (female negative, male positive); 2. Men and women 22–38 years old; 3. Males are clinically stable, failing to detect viral load of <75 copies/mL; screening for CD4 counts > 250; at least in the past 12 months with a history of continuous antiretroviral therapy; 4. Clinically confirmed to meet the medical guidelines for IVF therapy; 5. Fully informed consent of the couple to understand the purpose, risks and benefits of the trial; 6. Both subjects are willing to commit to using preventive contraception or maintaining abstinence for at least two months before egg collection and within one month after birth. 

排除标准:

父亲在精子收集前可检测到的病毒载量> 75拷贝/ mL 母亲或父亲在CRISPR / Cas9基因编辑的靶序列内具有遗传变异 母亲或父亲都有遗传变异,为CCR5靶向基因编辑创造了一种新颖的、高概率的脱靶位点 在该研究期间,在两次卵母细胞取出周期后自然怀孕或受精失败 先前多次(IVF)体外受精尝试失败 禁忌使用孕期禁忌药品 患有内分泌相关疾病,性激素处于异常水平 目前用使用放化疗药物治疗肿瘤相关疾病的 参与或最近参加了另一项使用研究性诊断测试,药物或设备的临床试验 患有疾病的受试者,包括酒精滥用或精神疾病,可能会导致研究人员或临床医师的判断而影响研究方案 

Exclusion criteria:

1. The viral load of the father before sperm collection is > 75 copies / mL; 2. Mother or father has genetic variation within the target sequence of the CRISPR/Cas9 gene editing; 3. Mother or father has genetic variation, creating a novel, high-probability off-target site for CCR5-targeted gene editing; 4. During the study, natural pregnancy or fertilization failed after two oocyte stimulation cycles; 5. Previously multiple in vitro fertilization (IVF) attempts failed; 6. Contraindications to use drugs during pregnancy; 7. With endocrine-related diseases, sexual hormones are at abnormal levels; 8. Currently using chemoradiotherapy drugs to treat tumor-related diseases; 9. Participated in or recently participated in another clinical trial using a research diagnostic test, drug or device; 10. Subjects with other diseases, including alcohol abuse or mental illness, that may influence the current protocol based on the researcher or clinician's judgment. 

研究实施时间:

Study execute time:

From2017-03-07To 2019-03-07 

干预措施:

Interventions:

组别:

Case series

样本量:

20

Group:

Case series

Sample size:

干预措施:

CCR5基因编辑

干预措施代码:

Intervention:

CCR5 gene editing

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):

 

Country:

China 

Province:

Guangdong 

City:

 

单位(医院):

深圳和美妇儿医院 

单位级别:

 

Institution
hospital:

Shenzhen HOME Women's and Children's Hospital  

Level of the institution:

 

测量指标:

Outcomes:

指标中文名:

怀孕并保证一个或多个活产

指标类型:

主要指标 

Outcome:

Pregnancy and guarantee one or more live births

Type:

Primary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

亲代和子代全基因组深度测序分析

指标类型:

主要指标 

Outcome:

Father, mother and progeny genome-wide deep sequencing analysis

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:

血液

组织:

Sample Name:

BLOOD

Tissue:

人体标本去向

使用后销毁 

说明

Fate of sample:

Destruction after use 

Note:

标本中文名:

胚胎

组织:

Sample Name:

EMBRYOS

Tissue:

人体标本去向

其它 

说明

Fate of sample:

Others 

Note:

征募研究对象情况:

Recruiting status:

暂停或中断

Suspending

年龄范围:

Participant age:

最小 Min age 22 years
最大 Max age 38 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):

HIV公益组织派发问卷调查,寻找满足条件志愿者,入组面谈,医疗结构体检,签署知情同意书随机入组

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

AIDS public welfare organizations randomly distributes questionnaires to find qualified volunteers, recruitment interviews, medical examinations at hospitals, and signing informed consent.

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):

试验结束6个月后提供网址和数据

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

Provide URL and data after six months of trial

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:

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),试验结束6个月后提供网址和数据

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

Clinical trial data will be collected using the case report form (CRF). The data will be managed by an electronic data capture (EDC) system. Results will be reported 6 months after the study completes.

数据管理委员会:

Data Managemen Committee:

有/Yes

注册人:

Name of Registration:

 2018-11-08
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