Clinical trial NCT03201848: The Efficacy and Safety of Huaiqihuang Granule in Children With Chronic Primary Immune Thrombocytopenia
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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE4
Conditions: Immune Thrombocytopenia
Keywords: Huaiqihuang granule
immune thrombocytopenia
randomized
double-blind
placebo-controlled,
Lead sponsor: Qidong Gaitianli Medicines Co., Ltd
Sponsor class: INDUSTRY
Collaborators: name: Huazhong University of Science and Technology
First posted: 2017-06-28
Enrollment: 216
Sex: ALL
Minimum age (years): 1
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

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).

Eligibility criteria: Inclusion Criteria:

1. Male or female, aged between 1 and 14 years (inclusive) ;
2. 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...
3. Meet the diagnosis criteria for Qi Yin deficiency in traditional Chinese medicine;
4. Time since initial diagnosis of ITP until randomization is at least 12 month, i.e., course of disease ≥1 year;
5. Values of prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT) are within 1.2 times of normal reference range;
6. 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;
7. 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:

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

Primary outcomes:
measure: the proportion of subjects whose hemorrhage has improved (clinical effective rate)
timeFrame: 24、48 and 60 Weeks after treatment
Secondary outcomes:
measure: the recovery rate of immune function
timeFrame: 24、48 and 60 Weeks after treatment
measure: the recovery rate of thyroid function
timeFrame: 12、24、36、48、60 and 72 Weeks after treatment
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Guangzhou Women and Children's Medical Center
state: Guangdong
city: Guangzhou
country: China
facility: The First Affiliated Hospital, Sun Yat-sen University
state: Guangdong
city: Zhengzhou
country: China
facility: The First Affiliated Hospital of Zhengzhou University
state: Henan
city: Wuhan
country: China
facility: Union Hospital of Tongji Medical College, Huazhong University of Science & Technology
state: Hubei
city: Wuhan
country: China
facility: Wuhan Children's Hospital
state: Hubei
city: Nanjing
country: China
facility: Children's Hospital of Nanjing Medical University
state: Jiangsu
city: Suzhou
country: China
facility: Children's Hospital of Soochow University
state: Jiangsu
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
state: Jiangxi
city: Qingdao
country: China
facility: The Affiliated Hospital of Qingdao University
state: Shandong
city: Luzhou
country: China
facility: The Affiliated Hospital of Southwest Medical University
state: Sichuan
city: Ürümqi
country: China
facility: First Affiliated Hospital of Xinjiang Medical University
state: Xinjiang
city: Beijing
country: China
facility: Beijing Children's Hospital. Capital Medical University
city: Beijing
country: China
facility: Children's Hospital Capital Institute of Pediatrics
city: Shanghai
country: China
facility: Xinhua Hospital Affiliated to Shanghai Jiaotong University School of Medicine
Study officials:
affiliation: Wuhan Union Hospital, China
name: Runming Jin, Professor
role: PRINCIPAL_INVESTIGATOR
References: