Clinical trial NCT05392647: 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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Myelodysplastic Syndromes
Keywords: hetrombopag
thrombocytopenia
Myelodysplastic Syndromes
Lead sponsor: Tongji Hospital
Sponsor class: OTHER
First posted: 2022-05-26
Enrollment: 28
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Platelet response
timeFrame: 24 weeks
Secondary outcomes:
measure: Overall response rate
timeFrame: 24 weeks
measure: Proportion of patients with platelet response during treatment
timeFrame: 24 weeks
measure: Incidence and severity of bleeding events according to the WHO Bleeding Scale
timeFrame: 28 weeks
measure: Adverse events
timeFrame: 28 weeks
Other outcomes:
Locations:
Study officials:
affiliation: Tongji Hospital
name: Jia Wei, PhD
role: PRINCIPAL_INVESTIGATOR
References: