A Clinical Study on the Efficacy and Safety of Hetrombopag in the Treatment of Thrombocytopenia Induced by Gynecological Tumor Therapy

The study consists of five sequential periods:

• Screening (≤ 4 weeks)
• Correction phase
• Maintenance phase (first treatment cycle, Cycle 1)
• Observation phase (second treatment cycle, Cycle 2)

• Follow-up Eligible subjects are gynaecological-cancer patients who developed ≥ grade-2 cancer-therapy-induced thrombocytopenia (CTIT) after anti-tumour therapy, i.e. platelet count < 75 × 10⁹/L.

  • Stage 1** - Prospective, multicentre, randomised clinical study (to inform the confirmatory stage 2) **Cohort 1 (n = 30)** Correction: hetrombopag 7.5 mg p.o. daily until PLT ≥ 100 × 10⁹/L. Within 24 h after completion of Cycle 1 anti-cancer therapy (end of chemotherapy if combined; otherwise end of investigational product on that day) restart hetrombopag 7.5 mg p.o. daily and continue until the last day of Cycle 1 (C1D21).

**Cohort 2 (n = 30)** Correction: hetrombopag 7.5 mg p.o. daily until PLT ≥ 100 × 10⁹/L. During Cycle 1, monitor PLT closely; if PLT falls < 75 × 10⁹/L, initiate hetrombopag 7.5 mg p.o. daily and continue until C1D21.

**Stage 2** - Prospective, multicentre, randomised, controlled clinical study *(preliminary design; sample size and details will be refined after stage-1 results)* **Experimental arm (n = 92)** Correction: hetrombopag 7.5 mg p.o. daily until PLT ≥ 100 × 10⁹/L. Restart within 24 h after completion of Cycle 1 therapy and continue daily until C1D21.

**Control arm (n = 92)** Correction: hetrombopag 7.5 mg p.o. daily until PLT ≥ 100 × 10⁹/L. During Cycle 1, monitor PLT; if PLT < 75 × 10⁹/L, initiate hetrombopag 7.5 mg p.o. daily until C1D21.

**Correction-phase rules** Discontinue hetrombopag once PLT ≥ 100 × 10⁹/L. If after ≥ 14 days of hetrombopag correction PLT remains < 100 × 10⁹/L and the next treatment cycle has not started, the investigator may resume hetrombopag or institute alternative platelet-enhancing rescue therapy at their discretion.

Study Overview

• Status: Not yet recruiting
• Conditions: Chemotherapy Induced Thrombocytopenia
• Intervention / Treatment: Drug: hetrombopag 7.5 mg orally once daily

• Study Type: Interventional
• Enrollment (Estimated): 244
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Huaiwu Lu; Phone Number: 02081332587; Email: luhuaiwu@mail.sysu.edu.cn
• Study Contact Backup: Name: Huaiwu Lu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Key eligibility criteria:

1. Female, aged 18-75 years (inclusive).
2. Histopathologically or cytologically confirmed ovarian, cervical, endometrial, or other gynecologic malignancy.
3. Thrombocytopenia (platelet count < 75 × 10⁹/L) attributable to anti-cancer therapy, with ≥ 2 further treatment cycles planned.
4. ECOG performance status 0-1.
5. Estimated life expectancy ≥ 12 weeks.
6. Women of child-bearing potential must have a negative serum pregnancy test within 7 days before first dose, not be breastfeeding, and agree to use effective contraception from study entry through 7 days after the last dose.
7. Able to provide written informed consent and comply with study procedures.

Exclusion criteria:

1. Pregnant or lactating women.
2. Unable to understand the study nature or give informed consent.
3. History of any arterial or venous thrombosis (stroke, TIA, MI, DVT, PE) or clinical/laboratory evidence of thrombophilia.
4. Cardiac disease within 3 months before screening: NYHA class III/IV heart failure, symptomatic arrhythmia requiring therapy, MI, arrhythmias increasing thrombotic risk (e.g., atrial fibrillation), or QTc prolongation.
5. Thrombocytopenia not related to anti-cancer therapy, active severe bleeding, or refractory persistent thrombocytopenia.

6. Significant hepatic impairment:

   • No liver metastases: ALT/AST > 3 × ULN or TBL > 3 × ULN
   • Liver metastases: ALT/AST ≥ 5 × ULN or TBL ≥ 5 × ULN
7. Known or suspected hypersensitivity/intolerance to TPO-receptor agonists or hetrombopag excipients.
8. Concomitant use of agents that may affect platelet count (e.g., Chinese herbal medicines, other thrombopoietic agents, antiplatelet drugs).
9. Receipt within 1 month before screening of TPO-RAs (eltrombopag, romiplostim, etc.), rhTPO, or rhIL-11.
10. Platelet transfusion within 3 days before randomisation/first dose.
11. Any condition that, in the investigator's opinion, renders the patient unsuitable for the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Correction phase: start hetrombopag 7.5 mg orally once daily and continue until platelet count norma	Drug: hetrombopag 7.5 mg orally once daily; Correction phase: start hetrombopag 7.5 mg orally once daily and continue until platelet count normalizes (PLT ≥ 100 × 10⁹/L). Within 24 h after completion of Cycle 1 anti-cancer therapy-defined as the end of chemotherapy infusion if on a combined regimen, or the end of study drug administration on that day for non-chemotherapy regimens-restart hetrombopag 7.5 mg orally once daily and maintain until the last day of Cycle 1 (C1D21).
Other: Correction phase: initiate hetrombopag 7.5 mg orally once daily and continue until platelet count re	Drug: hetrombopag 7.5 mg orally once daily; Correction phase: start hetrombopag 7.5 mg orally once daily and continue until platelet count normalizes (PLT ≥ 100 × 10⁹/L). Within 24 h after completion of Cycle 1 anti-cancer therapy-defined as the end of chemotherapy infusion if on a combined regimen, or the end of study drug administration on that day for non-chemotherapy regimens-restart hetrombopag 7.5 mg orally once daily and maintain until the last day of Cycle 1 (C1D21).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of subjects achieving a treatment response	Treatment-response criteria (all must be met): Completed the entire first treatment cycle (Cycle 1) with a platelet count ≥ 100 × 10⁹/L at C1D21 (window: +4 days).; Received no rescue therapy for thrombocytopenia (e.g., platelet transfusion, rhIL-11, rhTPO) from the start of Cycle 1 through C1D21 (window: +4 days).; Had no Cycle 2 modifications attributable to CTIT: no delay ≥ 4 days, no dose reduction ≥ 15 %, and no treatment suspension.	At the end of the first cycle (each cycle is 28 days).

Collaborators and Investigators

• Sponsor: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
• Investigators: Principal Investigator: Huaiwu Lu, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

Study record dates

Study Major Dates

• Study Start (Estimated): April 15, 2026
• Primary Completion (Estimated): April 15, 2027
• Study Completion (Estimated): June 30, 2027

Study Registration Dates

• First Submitted: January 21, 2026
• First Submitted That Met QC Criteria: February 12, 2026
• First Posted (Actual): February 13, 2026

Study Record Updates

• Last Update Posted (Actual): February 13, 2026
• Last Update Submitted That Met QC Criteria: February 12, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: hetrombopag
• Other Study ID Numbers: SYSKY-2025-1015-01

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