Comparison of Etamsylate Versus Placebo to Prevent Bleeding in HSCT

Comparison of Etamsylate Versus Placebo to Prevent Bleeding in Hematopoietic Stem Cell Transplantation Recipients：a Randomized, Double-blind, Phase 3, Clinical Study

This study employs a prospective, randomized, double-blind, placebo-controlled design. It aims to compare the efficacy of etamsylate versus placebo in preventing bleeding complications in patients with thrombocytopenia following hematopoietic stem cell transplantation.

Study Overview

• Status: Recruiting
• Conditions: Thrombocytopenia; Hematopoietic Stem Cell Transplantation
• Intervention / Treatment: Drug: etamsylate; Drug: Placebo

Detailed Description

This study employs a prospective, randomized, double-blind, placebo-controlled design. It aims to compare the efficacy of etamsylate versus placebo in preventing bleeding complications in patients with thrombocytopenia following hematopoietic stem cell transplantation. The primary endpoint is the incidence of death or bleeding events (WHO grade ≥2) within 30 days. Secondary endpoints focus on the safety profile of etamsylate versus placebo.

• Study Type: Interventional
• Enrollment (Estimated): 404
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Yi Luo; Phone Number: 86-13666609126; Email: luoyijr@zju.edu.com
• Study Contact Backup: Name: Luxin Yang; Phone Number: 86-13588756650; Email: yangluxin2016@163.com

Study Locations

• China
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Recruiting
      • The First Affiliated Hospital, College of Medicine, Zhejiang University
      • Contact: Luxin Yang; Phone Number: 86-13588756650; Email: yangluxin2016@163.com
      • Contact: Yi Luo; Phone Number: 86-13666609126; Email: luoyijr@zju.edu.cn

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Age between 18 and 70 years (inclusive), regardless of gender;
2. Patients diagnosed with a hematological disease requiring hematopoietic stem cell transplantation;
3. Expected platelet count ≤ 10 x 10⁹/L persisting for 5 days or more;
4. Normal coagulation function;
5. Adequate organ function: Aspartate Aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 3 times the Upper Limit of Normal (ULN), Total Bilirubin ≤ 2 x ULN; Serum Creatinine ≤ 2 x ULN; Creatinine Clearance ≥ 50 mL/min (calculated using the Cockcroft-Gault formula); Left Ventricular Ejection Fraction (LVEF) ≥ 50% as measured by Echocardiography (ECHO);
6. Voluntary provision of signed informed consent, with the ability to understand and comply with all study requirements.

Exclusion Criteria:

1. Diagnosis of acute promyelocytic leukemia confirmed according to WHO diagnostic criteria;
2. Pregnant or breastfeeding women, and women of childbearing potential unwilling to use effective contraception;
3. Presence of active bleeding or infection;
4. History of diagnosed primary immune thrombocytopenia or hemolytic uremic syndrome;
5. Patients with known hereditary or acquired hemorrhagic disorders;
6. Patients receiving anticoagulant or antiplatelet therapy;
7. Patients with severe cardiac insufficiency (left ventricular ejection fraction [EF] < 60%); or severe arrhythmias: history of clinically significant QTc prolongation (male > 450 ms; female > 470 ms), ventricular tachycardia, atrial fibrillation, second-degree heart block; myocardial infarction within 1 year prior to enrollment; or symptomatic coronary artery disease requiring medication; patients with severe liver impairment (liver function indices [ALT, TBIL] > 3 times the upper limit of normal [ULN]);
8. Patients with severe pulmonary insufficiency (obstructive and/or restrictive ventilatory defects);
9. Patients with severe renal insufficiency (renal function index [Cr] > 2 times ULN); or 24-hour urinary creatinine clearance rate (Ccr) < 50 ml/min;
10. Patients with mental disorders or other conditions preventing provision of informed consent and compliance with study treatment and procedural requirements;
11. Other reasons deemed by the investigator to make the patient ineligible for inclusion.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: etamsylate; Etamsylate Injection: Administer by intravenous infusion, diluted with 100ml of 0.9% Sodium Chloride Injection, twice daily.	Drug: etamsylate; etamsylate Injection, administer by intravenous infusion, diluted with 100ml of 0.9% Sodium Chloride Injection, twice daily.
Placebo Comparator: placebo; 0.9% Sodium Chloride Injection, 100 ml, twice daily, intravenous drip	Drug: Placebo; 0.9% Sodium Chloride Injection, 100 ml, twice daily, intravenous drip

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
WHO grade 2 or higher bleeding events or death.	The incidence of WHO grade 2 or higher bleeding events or death within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the incidence in the experimental group is 14.2% lower than that in the placebo group.	30 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The number of days without WHO grade 2 or higher bleeding events	The number of days without WHO grade 2 or higher bleeding events within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the number of days without WHO grade 2 or higher bleeding events is 2 days more in the experimental group than in the placebo group.	30 days
The number of platelet transfusions		The number of platelet transfusions within 30 days of medication administration. Etamsylate will be considered effective compared to placebo when the number of platelet transfusions in the experimental group is 2.5 fewer than in the placebo group.

Collaborators and Investigators

• Sponsor: First Affiliated Hospital of Zhejiang University
• Investigators: Principal Investigator: Yi Luo, The First Affiliated Hospital, College of Medicine, Zhejiang University Principal Investigator

Study record dates

Study Major Dates

• Study Start (Actual): December 16, 2025
• Primary Completion (Estimated): October 31, 2027
• Study Completion (Estimated): October 31, 2027

Study Registration Dates

• First Submitted: February 25, 2026
• First Submitted That Met QC Criteria: February 25, 2026
• First Posted (Actual): March 2, 2026

Study Record Updates

• Last Update Posted (Actual): March 2, 2026
• Last Update Submitted That Met QC Criteria: February 25, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: thrombocytopenia; bleeding events; etamsylate; Hematopoietic Stem Cell Transplantation recipients
• Additional Relevant MeSH Terms: Cytopenia; Hematologic Diseases; Blood Platelet Disorders; Hemic and Lymphatic Diseases; Thrombocytopenia; Sulfur Compounds; Organic Chemicals; Hydrocarbons; Hydrocarbons, Cyclic; Hydrocarbons, Aromatic; Benzene Derivatives; Sulfonic Acids; Sulfur Acids; Benzenesulfonates; Arylsulfonates; Arylsulfonic Acids; Ethamsylate
• Other Study ID Numbers: ZJU-HSCT-ETA

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