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

• Status: Not yet recruiting
• Trial end date: 2024-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Tongji Hospital

Criteria

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.