ITP: The Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia

Sponsor

Qidong Gaitianli Medicines Co., Ltd (Industry)

Overall Status

Recruiting

CT.gov ID

NCT03201848

Collaborator

Huazhong University of Science and Technology (Other)

216

Enrollment

Locations

Arms

63.5

Anticipated Duration (Months)

15.4

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a multisite, double-blind, randomized, placebo-controlled, and parallel study designed to evaluate the clinical efficacy and safety of Huaiqihuang granule for treatment to children with chronic primary immune thrombocytopenia (Qi Yin deficiency).

Condition or Disease	Intervention/Treatment	Phase
Immune Thrombocytopenia	Drug: Placebo Drug: Huaiqihuang Granule	Phase 4

Detailed Description

The randomized clinical trial included subjects (n =216) will be randomly divided into experimental (n = 162) and control groups (n =54) according to a random number table. Patients in the experimental group will receive Huaiqihuang Granule . Patients in the control group will receive placebo a placebo-controlled designed is used for this study. To minimize the risks, treatment stopping rules and subsequent measures are established for subjects whose disease under study become worsening during the study. Subjects receiving placebo treatment will switch to investigational drug after Week 24 to reduce the risk of lack of efficacy by placebo.

The primary outcome measures is proportion of patients whose hemorrhage has improved (clinical effective rate).

Study Design

Study Type:

Interventional

Anticipated Enrollment :

216 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

A Multisite, Randomized, Double-Blind, Placebo-Controlled, and Parallel Study to Evaluate the Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia (Qi Yin Deficiency)

Actual Study Start Date :

Nov 16, 2017

Anticipated Primary Completion Date :

Dec 3, 2022

Anticipated Study Completion Date :

Mar 3, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Huaiqihuang Granule Huaiqihuang Granule given to subject will be adjusted by body weight with treatment duration for 48 weeks	Drug: Huaiqihuang Granule Huaiqihuang Granule given to subject will be adjusted by body weight (<10kg, half a bag, 2 times a day; 10-20kg; 1 bag, 2 times a day; 20-30kg, 1.5 bag, 2 times a day; 30-50kg, 2 bags, 2 times a day; ≥50kg, 3 bags, 2 times a day) Other Names: Huaiqihuang
Placebo Comparator: Placebo Placebo given to subject will be adjusted by body weight After 24 weeks of placebo, change to Huaiqihuang Granule for another 24 weeks.	Drug: Placebo Placebo given to subject will be adjusted by body weight (<10kg, Placebo given to subject will be adjusted by body weight(<10kg, half a bag, 2 times a day; 10-20kg; 1 bag, 2 times a day; 20-30kg, 1.5 bag, 2 times a day; 30-50kg, 2 bags, 2 times a day; ≥50kg, 3 bags, 2 times a day;) . Drug: Huaiqihuang Granule Huaiqihuang Granule given to subject will be adjusted by body weight (<10kg, half a bag, 2 times a day; 10-20kg; 1 bag, 2 times a day; 20-30kg, 1.5 bag, 2 times a day; 30-50kg, 2 bags, 2 times a day; ≥50kg, 3 bags, 2 times a day) Other Names: Huaiqihuang

Arm

Intervention/Treatment

Experimental: Huaiqihuang Granule

Huaiqihuang Granule given to subject will be adjusted by body weight with treatment duration for 48 weeks

Drug: Huaiqihuang Granule

Huaiqihuang Granule given to subject will be adjusted by body weight (<10kg, half a bag, 2 times a day; 10-20kg; 1 bag, 2 times a day; 20-30kg, 1.5 bag, 2 times a day; 30-50kg, 2 bags, 2 times a day; ≥50kg, 3 bags, 2 times a day)

Other Names:

Huaiqihuang

Placebo Comparator: Placebo

Placebo given to subject will be adjusted by body weight After 24 weeks of placebo, change to Huaiqihuang Granule for another 24 weeks.

Drug: Placebo

Placebo given to subject will be adjusted by body weight (<10kg, Placebo given to subject will be adjusted by body weight(<10kg, half a bag, 2 times a day; 10-20kg; 1 bag, 2 times a day; 20-30kg, 1.5 bag, 2 times a day; 30-50kg, 2 bags, 2 times a day; ≥50kg, 3 bags, 2 times a day;) .

Drug: Huaiqihuang Granule

Other Names:

Huaiqihuang

Outcome Measures

Primary Outcome Measures

the proportion of subjects whose hemorrhage has improved (clinical effective rate) [24、48 and 60 Weeks after treatment]
The improvement of hemorrhage is defined if at least one of the following criteria is met: Severity of hemorrhage decreases one or more grade, the severity of hemorrhage is defined as following: none: there is any hemorrhage manifestation; mild: simple (often related with an unnoticeable minor collision without obvious ecchymosis) or obvious ecchymosis by collision; moderate: mucous membrane hemorrhage (nose hemorrhage, gum, oral mucosa) and menorrhagia; and severe: organ hemorrhage (digestive tract, respiratory tract, intracranial, etc.). Significant increase in platelet count defined as platelet count is over 30×109/L and increases more than twice compared with the baseline. The results must be tested at least twice with at least 7 days apart). The score of quality of life improves one or more points.

Secondary Outcome Measures

the recovery rate of immune function [24、48 and 60 Weeks after treatment]
The recovery rate of immune function is the proportion of subjects who have normalized immune function after treatment. The parameters of immune function include cytokines, lymphocyte subsets and auto immunity. One of the three tests has been restored to normal is defined as the immune function has returned to normal.
the recovery rate of thyroid function [12、24、36、48、60 and 72 Weeks after treatment]
The recovery rate of thyroid function is the proportion of subjects who have normalized immune function after treatment.

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 14 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male or female, aged between 1 and 14 years (inclusive) ;
Is diagnosed as immune thrombocytopenia according to the criteria of ICD-10, i.e., platelet < 100×109/L for at least two hematology tests with normal blood cell morphology; skin petechia, ecchymosis and (or) the clinical manifestations of mucosa and visceral hemorrhage; no splenomegaly; exclusion of other secondary thrombocytopenia, such as aplastic leukemia, aplastic anemia characterized by thrombocytopenia as the first hematologic abnormality, hereditary thrombocytopenia, thrombocytopenia secondary to other autoimmune diseases, infections or drugs etc...
Meet the diagnosis criteria for Qi Yin deficiency in traditional Chinese medicine;
Time since initial diagnosis of ITP until randomization is at least 12 month, i.e., course of disease ≥1 year;
Values of prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT) are within 1.2 times of normal reference range;
Values of biochemistry parameters including creatinine, ALT, AST, glucose, total bilirubin are less than 1.2 time of the corresponding upper limit of normal range;
Has signed and dated the informed consent in volunteer by the subject and the subject's legal guardian(s) (for subject who is less than 8 years old, the informed consent will be signed and dated by the subject's legal guardian(s) in volunteer).

Exclusion Criteria:

Is diagnosed as congenital thrombocytopenia;
Is diagnosed as secondary thrombocytopenia;
Is diagnosed as non-immune thrombocytopenia;
Has medical history of diabetes;
In the judgement of Investigator, the subject is not appropriate to participate in this study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Guangzhou Women and Children's Medical Center	Guangzhou	Guangdong	China
2	The First Affiliated Hospital, Sun Yat-sen University	Guangzhou	Guangdong	China
3	The First Affiliated Hospital of Zhengzhou University	Zhengzhou	Henan	China
4	Union Hospital of Tongji Medical College, Huazhong University of Science & Technology	Wuhan	Hubei	China	430022
5	Wuhan Children's Hospital	Wuhan	Hubei	China
6	Children's Hospital of Nanjing Medical University	Nanjing	Jiangsu	China
7	Children's Hospital of Soochow University	Suzhou	Jiangsu	China
8	Jiangxi Provincial Children's Hospital	Nanchang	Jiangxi	China
9	The Affiliated Hospital of Qingdao University	Qingdao	Shandong	China
10	The Affiliated Hospital of Southwest Medical University	Luzhou	Sichuan	China
11	First Affiliated Hospital of Xinjiang Medical University	Ürümqi	Xinjiang	China
12	Beijing Children's Hospital. Capital Medical University	Beijing		China
13	Children's Hospital Capital Institute of Pediatrics	Beijing		China
14	Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine	Shanghai		China