Hetrombopag for Lower-risk MDS With Thrombocytopenia: a Prospective，Open-label, Single-arm Study.

Study Overview
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Brief Summary
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The is a phase 2, single-arm, open-label clinical study assessing the efficacy and safety of hetrombopag to increase platelet count in lower-risk MDS patients with thrombocytopenia.

Detailed Description
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The is a phase 2, single-arm, open-label clinical study assessing the efficacy and safety of hetrombopag to increase platelet count in patients with very low, low, or intermediate risk myelodysplastic syndromes(MDS) according to the revised International Prognostic Scoring System (IPSS-R) with thrombocytopenia. The primary endpoint was proportion of patients achieving platelet response at week 24.

Official Title
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Hetrombopag for Lower-risk MDS With Thrombocytopenia: a Prospective，Open-label, Single-arm Study.

Conditions
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Myelodysplastic Syndromes

Intervention / Treatment
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* Drug: Hetrombopag


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 18 years or older； Confirmed diagnosis of MDS based on 2016 WHO (World Health Organization) and classification with very low, low or intermediate (score ≤ 3.5) risk according to the revised International Prognostic Scoring System (IPSS-R). Platelets < 30 x 10^9/L or platelets < 50 x 10^9/L with active bleeding at screening. Eastern Cooperative Oncology Group (ECOG) performance score ≤ 2； Ability to understand information material and written informed consent. Exclusion Criteria: Severe active bleeding/infection or any other uncontrolled severe condition; History of treatment with thrombopoietin receptor agonists (eltrombopag, avatrombopag, romiplostim, etc.) or recombinant human thrombopoietin with 1 month prior; Patients with known active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) at screening（If hepatitis B surface antigen or hepatitis B core antibody are positive, HBV-DNA testing are needed. If positive, it suggests a viral replication and subjects will be excluded. If hepatitis C antibody are positive, HCV-RNA testing are needed. If positive, it suggests a viral replication and subjects will be excluded）; Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2.5 x upper limit of normal (ULN), total bilirubin >2.5 x ULN, creatinine >1.5 x ULN; History of portal hypertension or cirrhosis； History of any types of solid tumors within 5 years prior, whether the tumors has been treated, metastasized or relapsed (except basal cell carcinoma); History of congestive heart failure requiring medical management, cardiac arrhythmias, peripheral arteriovenous thrombosis within 1 years prior, or myocardial infarction or cerebral infarction within 3 months prior; ECOG performance score ≥ 3; Female subjects who are pregnant or breastfeeding, or subjects who are planning pregnancy within 6 months; Women of child-bearing potential and men must agree to use contraception prior to study entry and for the duration of study participation; Participated in other clinical trials within 3 months prior; Any condition which, in the investigator's opinion, deems the subject an unsuitable candidate to receive study drug.

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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Single arm<br>Hetrombopag | Drug: Hetrombopag<br>* All eligible patients were given hetrombopag orally at an initial dose of 5 mg once daily after overnight fasting (>8 hours of fasting) and at least 2 hours before a meal . The dose of hetrombopag could be modified on the basis of the platelet count of patients after 2 weeks of administration.<br>* Other names: Hetrombopag Olamine Tablets;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Platelet response | proportion of patients achieving platelet responses at week 24 | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall response rate | Patients will be assessed for response according to the IWG criteria. | 24 weeks | 
| Proportion of patients with platelet response during treatment | Improvement in platelet counts by 24 weeks of therapy | 24 weeks | 
| Incidence and severity of bleeding events according to the WHO Bleeding Scale | The severity of bleeding symptoms was graded according to the World Health Organization (WHO) bleeding scale (grade 0: no bleeding; grade 1: petechiae; grade 2: mild blood loss; grade 3, gross blood loss; grade 4: debilitating blood loss). | 28 weeks | 
| Adverse events | Adverse events were recorded and graded according to National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0 | 28 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hetrombopag, thrombocytopenia, Myelodysplastic Syndromes