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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PartA, open-label | Experimental | Hetrombopag plus standard of care |
|
| PartB, double-blind treatment group | Experimental | Hetrombopag plus standard of care |
|
| Placebo Comparator | Placebo Comparator | Placebo plus standard of care Part B, double-blind treatment group |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hetrombopag | Drug | Thrombopoietin receptor agonist |
|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Beijing children's hospital .Capital medical university | Beijing | Beijing Municipality | 100032 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41952212 | Derived | Wu R, Gao J, Liu Y, Yu J, Hu S, Liang H, Jiang H, Zhai X, Zhou M, Hao W, Mei H, Li F, Chen Y, Liu W, Yan M, Li R, Xiong J, Zhu Z, Cheng X, Wang T. Efficacy and safety of hetrombopag, a novel thrombopoietin receptor agonist, in children and adolescents with immune thrombocytopenia: results from a randomized, multicenter, placebo-controlled phase 3 trial. J Hematol Oncol. 2026 Apr 8;19(1):24. doi: 10.1186/s13045-026-01790-x. |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C000614661 | hetrombopag |
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Parallel assignment
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| Placebo | Drug | Placebo with no active pharmaceutical ingredient |
|
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |