Etamsylate versus placebo to prevent bleeding after stem cell transplant

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

This will test whether etamsylate can reduce bleeding in adults with very low platelets after hematopoietic stem cell transplantation.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 404 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: First Affiliated Hospital of Zhejiang University (Academic / other)
• Locations: 1 site (Hangzhou, Zhejiang)
• Trial ID: NCT07442513 on ClinicalTrials.gov

What this trial studies

This phase 3, randomized, double‑blind, placebo‑controlled trial compares etamsylate to placebo in adults who develop thrombocytopenia after hematopoietic stem cell transplantation. Eligible participants are 18–70 years old with platelet counts ≤10×10^9/L persisting for at least five days and adequate organ and coagulation function. The primary endpoint is the incidence of death or WHO grade ≥2 bleeding within 30 days, with secondary endpoints focused on safety and adverse events. The trial is conducted at The First Affiliated Hospital, College of Medicine, Zhejiang University in Hangzhou, China.

Who should consider this trial

Good fit: Adults aged 18–70 undergoing hematopoietic stem cell transplantation who have persistent severe thrombocytopenia (platelets ≤10×10^9/L for ≥5 days) with normal coagulation and adequate organ function.

Not a fit: Patients outside the age range, those with acute promyelocytic leukemia, pregnant or breastfeeding women, or those without persistent severe thrombocytopenia are unlikely to receive benefit from this intervention.

Why it matters

Potential benefit: If successful, etamsylate could lower the risk of bleeding complications and related deaths in patients with severe thrombocytopenia after stem cell transplant.

How similar studies have performed: Etamsylate has been used as a hemostatic agent in other bleeding contexts with mixed or limited evidence, and its benefit specifically for post‑HSCT thrombocytopenia is not well established.

Eligibility criteria

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.

Where this trial is running

Hangzhou, Zhejiang

• The First Affiliated Hospital, College of Medicine, Zhejiang University — Hangzhou, Zhejiang, China (Recruiting)

Study contacts

• Principal investigator: Yi Luo — The First Affiliated Hospital, College of Medicine, Zhejiang University Principal Investigator
• Study coordinator: Yi Luo

Conditions Hematopoietic Stem Cell Transplantation; Thrombocytopenia; etamsylate; Hematopoietic Stem Cell Transplantation recipients; thrombocytopenia; bleeding events