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Effectiveness of Modified Buzhong Yiqi Decoction in treating myasthenia gravis: study protocol for a series of N-of-1 trial
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注册号:

Registration number:

ChiCTR2000040477 

最近更新日期:

Date of Last Refreshed on:

2021-07-01 

注册时间:

Date of Registration:

2020-11-29 

注册号状态:

预注册  

Registration Status:

Prospective registration  

注册题目:

加味补中益气汤治疗重症肌无力的疗效评价:系列单病例随机对照试验研究方案 

Public title:

Effectiveness of Modified Buzhong Yiqi Decoction in treating myasthenia gravis: study protocol for a series of N-of-1 trial 

注册题目简写:

 

English Acronym:

 

研究课题的正式科学名称:

中医药治疗重症肌无力的病因病机创新研究(N-of-1) 

Scientific title:

Innovative research on the pathogenesis of myasthenia gravis in traditional Chinese medicine (N-of-1) 

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:

ChiMCTR2000004105 

申请注册联系人:

翁森辉 

研究负责人:

刘凤斌 

Applicant:

Senhui Weng 

Study leader:

Fengbin Liu 

申请注册联系人电话:

Applicant telephone:

+86 18813966805 

研究负责人电话:

Study leader's telephone:

+86 18902296111 

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:

782106945@qq.com 

研究负责人电子邮件:

Study leader's E-mail:

liufb163@163.com 

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:

广州市机场路12号 

研究负责人通讯地址:

广州市机场路16号 

Applicant address:

12 Jichang Road, Baiyun District, Guangzhou, Guangdong, China 

Study leader's address:

16 Jichang Road, Baiyun District, Guangzhou, 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:

广州中医药大学 

Applicant's institution:

Guangzhou University of TCM.  

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:

ZYYECK【2019】119 

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:

广州中医药大学第一附属医院伦理委员会 

Name of the ethic committee:

Ethics Committee of The First Affiliated Hospital of Guangzhou University of Chinese Medicine 

伦理委员会批准日期:

Date of approved by ethic committee:

2019-12-09 

伦理委员会联系人:

黎欣盈 

Contact Name of the ethic committee:

Xinying Li 

伦理委员会联系地址:

广州中医药大学第一附属医院办公楼501 

Contact Address of the ethic committee:

Room 501, Administration Building, The First Affiliated Hospital of Guangzhou University of Chinese Medicine, 16 Jichang Road, Baiyun District, Guangzhou, 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:

广州中医药大学第一附属医院 

Primary sponsor:

The First Affiliated Hospital of Guangzhou University of Chinese Medicine 

研究实施负责(组长)单位地址:

广州市机场路16号 

Primary sponsor's address:

16 Jichang Road, Baiyun District, Guangzhou, Guangdong, China 

试验主办单位(项目批准或申办者):

Secondary sponsor:

经费或物资来源:

广州中医药大学科研团队培育项目 

Source(s) of funding:

the Scientific Research Team Training Project Of Guangzhou University of Chinese Medicine 

研究疾病:

重症肌无力 

Target disease:

myasthenia gravis 

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:

其它 

Study phase:

N/A 

研究目的:

探讨加味补中益气颗粒治疗MG患者的临床疗效,得出中医药治疗重症肌无力高质量临床证据。 

Objectives of Study:

To evaluate the effectiveness of MBYD in the treatment of MG and explore the clinical evidence of treating myasthenia gravis with Chinese medicine was obtained. 

药物成份或治疗方案详述:

 

Description for medicine or protocol of treatment in detail:

 

研究设计:

单病例随机对照研究 

Study design:

N of 1 Trial 

纳入标准:

1)患者年龄18 ~ 65岁,性别不限 2)根据《中国重症肌无力诊疗指南(2020)》诊断为重症肌无力患者:有典型的MG(波动性肌无力)临床特征,但未患其他疾病,且符合以下药理学检查、电生理特征或血清抗体检测三点之一的患者 3)根据《中医内科临床诊治指南:重症肌无力(2020)》纳入脾胃虚证或脾肾虚证患者。 4)美国重症肌无力(MGFA)临床分级为II级或III级,且MG稳定期至少3个月 5)定量重症肌无力(QMG)评分大于6分者 6)接受糖皮质激素治疗的患者每日服用强的松(或其他糖皮质激素等量)不宜超过15mg。 

Inclusion criteria

1) Patients aged between 18 to 65 years; 2) Patients diagnosed with MG based on The Chinese guidelines for the diagnosis and treatment of myasthenia gravis (2020): patients with typical clinical features of MG (fluctuating myasthenia) excluded from other diseases and meeting any of the following three points, including pharmacological examination, electrophysiological characteristics or serum antibody detection; 3) Patients attached to the spleen-stomach deficiency syndrome or spleen-kidney deficiency syndrome will be enrolled which is based on The guidelines for clinical diagnosis and treatment of internal medicine of traditional Chinese Medicine: myasthenia gravis (2020); 4) Patients identified as class II or III according to myasthenia gravis foundation of America (MGFA) clinical classification and are in the stable stage of MG at least 3 months; 5) Patients with Quantitative Myasthenia Gravis (QMG) score more than 6; 6) Patients treated with glucocorticoid administration should not take more than 15mg prednisone (or an equivalent dose of other glucocorticoids) per day. 

排除标准:

1)试验期间怀孕、哺乳期或有妊娠计划的女性患者 2)患有其他自身免疫性疾病(如多发性肌炎、多发性硬化症、类风湿性关节炎),可能影响评估和治疗的患者 3)严重的心、肾、肝、肺、血液系统、感染性疾病或癌症患者 4)神经精神障碍患者不能配合 5) 3个月内进行血浆置换、糖皮质激素或-球蛋白脉冲治疗 6)患者在半年内接受胸腺切除术 7)本试验中任何药物过敏者 8)参与其他试验的患者 9)患者前两个月服用过抗生素、益生菌或抗酸剂 

Exclusion criteria:

1) Female patients who are pregnant or lactating or have a pregnancy plan during the trial; 2) Patients with other autoimmune diseases (e.g., polymyositis, multiple sclerosis, rheumatoid arthritis) that may impact the assessment and treatment; 3) Patients with severe heart, kidney, liver, lung, hematological system, infectious diseases or cancer; 4) Patients with neuropsychiatric disorders that cannot cooperate; 5) Patients received plasma exchange, glucocorticoid or gamma-globin pulse therapy within 3 months; 6) Patients received thymectomy within half a year; 8) Patients participating in other trials; 9) Patients consumed antibiotics, probiotics or anti-acids during the previous two months. 

研究实施时间:

Study execute time:

From2020-12-05To 2022-05-30 

征募观察对象时间:

Recruiting time:

From2020-12-05To 2021-12-01 

干预措施:

Interventions:

组别:

西药加脾虚/脾肾两虚组或安慰剂组

样本量:

12

Group:

Experimental group

Sample size:

干预措施:

加味补中益气颗粒(健脾补肾升阳颗粒)

干预措施代码:

Intervention:

Modified Buzhong Yiqi Granule combined with Western medicine

Intervention code:

研究实施地点:

Countries of recruitment and research settings:

国家:

中国 

省(直辖市):

广东省 

市(区县):

广州市 

Country:

China 

Province:

Guangdong 

City:

Guangzhou 

单位(医院):

广州中医药大学第一附属医院 

单位级别:

三级甲等 

Institution
hospital:

The First Affiliated Hospital of Guangzhou University of Chiniese Medicine  

Level of the institution:

Tertiary A Hospital 

测量指标:

Outcomes:

指标中文名:

MGC

指标类型:

次要指标 

Outcome:

MGC

Type:

Secondary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

MGQOL

指标类型:

次要指标 

Outcome:

MGQOL

Type:

Secondary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

Treg和细胞因子水平IL-4/IL-17A,TGF-β,INF-γ

指标类型:

次要指标 

Outcome:

level of Treg cells and cytokines (IL-4, IL-17A, INF-γ, TGF-β)

Type:

Secondary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

副作用的减轻

指标类型:

次要指标 

Outcome:

reduction of the side effects

Type:

Secondary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

菌群改善

指标类型:

次要指标 

Outcome:

the composition of gut microbiota

Type:

Secondary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

QMG评分

指标类型:

主要指标 

Outcome:

QMG score

Type:

Primary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

MG-ADL

指标类型:

次要指标 

Outcome:

MG-ADL

Type:

Secondary indicator 

测量时间点:

测量方法:

Measure time point of outcome:

Measure method:

指标中文名:

MG-pro

指标类型:

次要指标 

Outcome:

MG-pro

Type:

Secondary 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:

粪便

组织:

Sample Name:

stool

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 18 years
最大 Max age 65 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):

n -1试验将包括三个周期,为期8周,QJC组(A)为4周,安慰剂组(B)为4周。每个周期只允许AB和BA,序列是AB还是BA取决于SAS随机规划。在两个治疗周期之间将有一个1周的洗脱期,以最小化每个治疗周期之间的转移影响,并允许我们对结果进行评分和记录。随机人:李景濠

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

Only AB and BA are allowed in each cycle and whether the sequence is AB or BA depends on the SAS random programme.

盲法:

未说明

Blinding:

Not stated

是否共享原始数据:

IPD sharing

Yes

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):

edc平台

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

edc

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:

电子采集和管理系统(Electronic Data Capture,EDC)

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

Electronic Data Capture,EDC

数据与安全监察委员会:

Data and Safety Monitoring Committee:

有/Yes

注册人:

Name of Registration:

 2020-11-29
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