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

NCT05392647 | Unknown Phase 2

• 1 other identifier: OBU-HuB-MDS-II-001
• Study Type: interventional
• Target: 28
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Myelodysplastic Syndromes

Keywords

hetrombopag; thrombocytopenia; Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (1)

• Platelet response

  proportion of patients achieving platelet responses at week 24

  24 weeks

Secondary Outcomes (4)

• Overall response rate

  24 weeks

• Proportion of patients with platelet response during treatment

  24 weeks

• Incidence and severity of bleeding events according to the WHO Bleeding Scale

  28 weeks

• Adverse events

  28 weeks

Study Arms (1)

Single arm

EXPERIMENTAL

Hetrombopag

Drug: Hetrombopag

Interventions

Hetrombopag 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.

Also known as: Hetrombopag Olamine Tablets

Single arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Tongji Hospital: lead

MeSH Terms

Conditions

Myelodysplastic Syndromes; Thrombocytopenia

Interventions

hetrombopag

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Cytopenia

Study Officials

• Jia Wei, PhD

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jia Wei, PhD

CONTACT

86-13986102084; jiawei@tjh.tjmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: associate chief physician

Study Record Dates

• First Submitted: May 23, 2022
• First Posted: May 26, 2022
• Study Start: July 1, 2022
• Primary Completion: July 30, 2024
• Study Completion: December 31, 2024
• Last Updated: May 26, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share