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

Phase 2

Not yet recruiting

• Conditions: Myelodysplastic Syndromes
• Interventions: Drug: Hetrombopag

• Registration Number: NCT05392647
• Lead Sponsor: Tongji Hospital

Brief Summary

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

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.

Study Timeline

• Status Verified: 2022-05
• Study First Submitted: 2022-05-23
• Study First Submitted QC: 2022-05-23
• Last Update Submitted: 2022-05-23
• Study First Posted: 2022-05-26
• Last Update Posted: 2022-05-26
• Study Start: 2022-07-01
• Primary Completion: 2024-07-30
• Study Completion: 2024-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 28

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Single arm	Hetrombopag	Hetrombopag

Primary Outcome Measures

Name	Time	Method
Platelet response	24 weeks	proportion of patients achieving platelet responses at week 24

Secondary Outcome Measures

Name	Time	Method
Adverse events	28 weeks	Adverse events were recorded and graded according to National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0
Overall response rate	24 weeks	Patients will be assessed for response according to the IWG criteria.
Proportion of patients with platelet response during treatment	24 weeks	Improvement in platelet counts by 24 weeks of therapy
Incidence and severity of bleeding events according to the WHO Bleeding Scale	28 weeks	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).