High-dose Dexamethasone Plus Hetrombopag vs High-dose Dexamethasone Alone as Frontline Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Prospective, Multicenter, Randomized Trial

Study Overview
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Brief Summary
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The project was undertaking by Qilu Hospital of Shandong University in China. In order to report the efficacy and safety of Hetrombopag plus high-dose dexamethasone for the treatment of adults with newly-diagnosed primary immune thrombocytopenia (ITP).

Detailed Description
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The investigators anticipate to undertaking a parallel group, randomised controlled trial of 100 ITP patients. One part of the participants are randomly selected to receive hetrombopag with starting dose 5mg po qd for 8 weeks（increase daily dose to a maximum of 7.5 mg/day if platelet count<50000 per μL following at least 2 weeks of treatment) combining with dexamethasone (given at a dose of 40 mg qd for 4 consecutive days). The others are selected to receive high-dose of dexamethasone alone. Patients who do not respond to dexamethasone may receive another cycle of high-dose dexamethasone therapy within 2 weeks. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study. The purpose of this study is to report the efficacy and safety of Hetrombopag combining with high-dose dexamethasone therapy for the treatment of newly diagnosed ITP.

Official Title
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High-dose Dexamethasone Plus Hetrombopag Versus High-dose Dexamethasone Alone as Frontline Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia (ITP):A Prospective Multicenter Randomized Trial

Conditions
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ITP - Immune Thrombocytopenia

Intervention / Treatment
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* Drug: hetrombopag 5mg po qd
* Drug: High-dose Dexamethasone


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Older than 18 years Meet the diagnostic criteria for newly diagnosed immune thrombocytopenia (diagnosed within 3 month); platelet count <30*10^9/L, or < 50*10^9/L with bleeding manifestations, both; Willing and able to sign written informed consent Exclusion Criteria: secondary thrombocytopenia or graded MF≥2 myelofbrosis based on the European Consensus Scale Previous history of treatment for ITP, except Platelet transfusion, ITP-directed Prednisone therapy no more than 2 weeks or TPO therapy no more than 1 week and stopped ≥1 week before randomization No response to TPO-RA or rhTPO HIV, hepatitis C or B virus infection pregnancy or lactation; arterial or venous thromboembolism within the 6 months before screening total bilirubinalanine, aminotransferase or aspartate transaminase>3×upper limit of normal (ULN), serum creatinine>1.5×ULN congestive heart failure (New York Heart Association [NYHA] class III/IV); neoplastic disease within the past 5 years; liver cirrhosis people who could not adhere to the protocol or were planning to have a surgical procedure in 6 months.

Ages Eligible for Study
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Minimum Age: 18 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
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Hetrombopag plus High-dose Dexamethasone<br>Hetrombopag 5mg po qd; HD-DEX 40mg qd for 4 days | Drug: hetrombopag 5mg po qd<br>* hetrombopag 5mg po qd for 8 weeks, combining with dexamethasone 40 mg qd for 4 days<br>Drug: High-dose Dexamethasone<br>* dexamethasone 40 mg qd for 4 days<br>|
| Active Comparator: High-dose Dexamethasone<br>HD-DEX 40mg qd for 4 days | Drug: High-dose Dexamethasone<br>* dexamethasone 40 mg qd for 4 days<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 26 week sustained overall response to ITP treatments | Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding. | 26-week after treatment started | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 28-day initial complete response to ITP treatment | Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding. | 28 days after treatment started] | 
| 28-day initial overall response to ITP treatment | Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding. | 28 days after treatment started | 
| 8-week complete response to ITP treatment | Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding. | 8 weeks after treatment started | 
| 8-week overall response to ITP treatment | No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding. | 8 weeks after treatment started | 
| time to response | the time from treatment initiation to achieve a complete response or a partial response | an average of 6 months | 
| duration of response | the time from achievement of a complete response or a partial response to the loss of response | through study completion, an average of one year | 
| therapy associated adverse events |  | through study completion, an average of one year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hetrombopag, Dexamethasone