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

Primary Purpose

Myelodysplastic Syndromes

Status

Not yet recruiting

Phase

Phase 2

Locations

Study Type

Interventional

Intervention

Hetrombopag

About this trial

This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring hetrombopag, thrombocytopenia, Myelodysplastic Syndromes

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single arm

Arm Description

Hetrombopag

Outcomes

Primary Outcome Measures

Platelet response

proportion of patients achieving platelet responses at week 24

Secondary Outcome Measures

Overall response rate

Patients will be assessed for response according to the IWG criteria.

Proportion of patients with platelet response during treatment

Improvement in platelet counts by 24 weeks of therapy

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).

Adverse events

Adverse events were recorded and graded according to National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0

Full Information

NCT ID

NCT05392647

First Posted

May 23, 2022

Last Updated

May 23, 2022

Sponsor

Tongji Hospital

1. Study Identification

Unique Protocol Identification Number

NCT05392647

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2022

Overall Recruitment Status

Not yet recruiting

Study Start Date

July 1, 2022 (Anticipated)

Primary Completion Date

July 30, 2024 (Anticipated)

Study Completion Date

December 31, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Tongji Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myelodysplastic Syndromes

Keywords

hetrombopag, thrombocytopenia, Myelodysplastic Syndromes

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

28 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Single arm

Arm Type

Experimental

Arm Description

Hetrombopag

Intervention Type

Drug

Intervention Name(s)

Hetrombopag

Other Intervention Name(s)

Hetrombopag Olamine Tablets

Intervention Description

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.

Primary Outcome Measure Information:

Title

Platelet response

Description

proportion of patients achieving platelet responses at week 24

Time Frame

24 weeks

Secondary Outcome Measure Information:

Title

Overall response rate

Description

Patients will be assessed for response according to the IWG criteria.

Time Frame

24 weeks

Title

Proportion of patients with platelet response during treatment

Description

Improvement in platelet counts by 24 weeks of therapy

Time Frame

24 weeks

Title

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

Description

Time Frame

28 weeks

Title

Adverse events

Description

Adverse events were recorded and graded according to National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0

Time Frame

28 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Jia Wei, PhD

Phone

86-13986102084

jiawei@tjh.tjmu.edu.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jia Wei, PhD

Organizational Affiliation

Tongji Hospital

Official's Role

Principal Investigator

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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