Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia

Study Overview
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Brief Summary
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The purpose of this study is to investigate the efficacy, safety of Hetrombopag in children with previously treated chronic immune thrombocytopenia who are between 6 and 17 years of age. This is a 2 part study. In part A, patients will receive Hetrombopag for 8 weeks. In part B, all patients will receive Hetrombopag for 24 weeks.

Detailed Description
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This is a two-part, double-blind, randomized, placebo-controlled, and open-label Phase III study to investigate the efficacy, safety of Hetrombopag in pediatric patients with previously treated chronic ITP. In Part A, patients will receive Hetrombopag for 8 weeks. After completing Part A, patients will begin Part B, in which they will be randomized to receive Hetrombopag or placebo in a 12 week double-blind, placebo-controlled treatment period, following an open-label 12 week treatment period.

Official Title
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Safety and Efficacy of Hetrombopag in Children and Adolescents With Chronic Primary Immune Thrombocytopenia：a Randomized, Multicenter, Placebo-controlled Trial

Conditions
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Immune Thrombocytopenia

Intervention / Treatment
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* Drug: Hetrombopag
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age ≥ 6 years old and ≤ 17 years old,both sexes. Part A:Confirmed diagnosis of ITP ≥6months; Part B: Confirmed diagnosis of ITP ≥12months;Platelets <30×10^9/L twice in a row,and platelets <30×10^9/L before taking the medicine. Subjects who are refractory or have relapsed after at least one prior ITP therapy. Birth control during and 28 days after the trial. Written informed consent must be obtained from the patient's guardian and accompanying informed assent from the patient (for children over 8 years old). Exclusion Criteria: No evidence of other causes of thrombocytopenia. Diagnosis as Evans or Wiskott-Aldrich comprehensive. Patients with any prior history of arterial or venous thrombosis, or diagnosis as thrombophilia. Suffering from serious, progressive, uncontrolled kidney, liver, gastrointestinal, endocrine, lung, heart, nervous system, brain, or mental illness. ALT, AST, or ALP> 1.5 x upper limit of normal (ULN), DBLI, or Scr > 1.2 x upper limit of normal (ULN). Active HIV or HCV-Ab positive,HBsAg positive. PT result exceeds normal by more than ±3s, APTT result exceeds normal by more than ±10s Participated in clinical trials of other drugs (received experimental drugs) within 3 months prior to medication. The inestigators determined that other conditions were inappropriate for participation in this clinical trial.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Parallel assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PartA, open-label<br>Hetrombopag plus standard of care | Drug: Hetrombopag<br>* Thrombopoietin receptor agonist<br>|
| Experimental: PartB, double-blind treatment group<br>Hetrombopag plus standard of care | Drug: Hetrombopag<br>* Thrombopoietin receptor agonist<br>|
| Placebo Comparator: Placebo Comparator<br>Placebo plus standard of care Part B, double-blind treatment group | Drug: Placebo<br>* Placebo with no active pharmaceutical ingredient<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| the main parameters in population PK/PD modeling in Part A | Peak Plasma Concentration (Cmax) | from baseline to Week 2 | 
| the proportion of patients with a platelet count ≥50×10^9/L at week 10. | efficacy in part B | from baseline to Week 10 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ITP