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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: Hetrombopag

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 Type: Interventional
• Enrollment (Anticipated): 28
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Jia Wei, PhD; Phone Number: 86-13986102084; Email: jiawei@tjh.tjmu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single arm; Hetrombopag	Drug: 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.; Other Names: Hetrombopag Olamine Tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet response	proportion of patients achieving platelet responses at week 24	24 weeks

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Tongji Hospital
• Investigators: Principal Investigator: Jia Wei, PhD, Tongji Hospital

Study record dates

Study Major Dates

• Study Start (Anticipated): July 1, 2022
• Primary Completion (Anticipated): July 30, 2024
• Study Completion (Anticipated): December 31, 2024

Study Registration Dates

• First Submitted: May 23, 2022
• First Submitted That Met QC Criteria: May 23, 2022
• First Posted (Actual): May 26, 2022

Study Record Updates

• Last Update Posted (Actual): May 26, 2022
• Last Update Submitted That Met QC Criteria: May 23, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Keywords: Myelodysplastic Syndromes; thrombocytopenia; hetrombopag
• Additional Relevant MeSH Terms: Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Blood Platelet Disorders; Precancerous Conditions; Myelodysplastic Syndromes; Preleukemia; Thrombocytopenia
• Other Study ID Numbers: OBU-HuB-MDS-II-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No