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

NCT ID: NCT05392647

Last Updated: 2022-05-26

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 28 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-07-01
• Study Completion Date: 2024-12-31

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.

Conditions

Myelodysplastic Syndromes

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Single arm

Hetrombopag

Group Type: EXPERIMENTAL

Hetrombopag

Intervention Type: DRUG

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.

Interventions

Hetrombopag

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.

Intervention Type: DRUG

Other Intervention Names

Hetrombopag Olamine Tablets

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tongji Hospital

OTHER

Sponsor Role: lead

Responsible Party

Jia Wei

associate chief physician

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Jia Wei, PhD

Role: PRINCIPAL_INVESTIGATOR

Tongji Hospital

Central Contacts

Jia Wei, PhD

Role: CONTACT

jiawei@tjh.tjmu.edu.cn

86-13986102084

Other Identifiers

OBU-HuB-MDS-II-001

Identifier Type: -

Identifier Source: org_study_id