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

Tongji Hospital

Status and phase

Not yet enrolling

Phase 2

Conditions

Myelodysplastic Syndromes

Treatments

Drug: Hetrombopag

Study type

Interventional

Funder types

Other

Identifiers

NCT05392647

OBU-HuB-MDS-II-001

Details and patient eligibility

About

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.

Full 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.

Enrollment

28 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.