Hetrombopag in Promoting Platelet Engraftment After Auto-HSCT in Children With Neuroblastoma

NCT06818916 | Recruiting

• 1 other identifier: [2024]-Y-002-D
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to learn if hetrombopag promotes platelet engraftment in neuroblastoma children undergoing autologous hematopoietic stem cell transplantation. The main question it aims to answer is: · Does drug hetrombopag promote platelet engraftment? Participants will take hetrombopag every day for 6 weeks or until platelet \> 100\*10\^9/L.

Conditions

Neuroblastoma; Delayed Platelet Engraftment; Autologous Hematopoietic Stem Cell Transplantation

Keywords

Hetrombopag; delayed platelet engraftment; neuroblastoma; pediatrics; autologous hematopoietic stem cell transplantation

Outcome Measures

Primary Outcomes (1)

• Accumulated platelet engraftment rate

  Platelet engraftment is defined as a continuous 7-day PLT ≥ 20 × 10\^9/L without platelet infusion.

  From enrollment to 60 days post-transplant

Secondary Outcomes (4)

• Number of platelet transfusions

  From enrollment to 60 days post-transplant

• Time to platelet engraftment

  From enrollment to 60 days post-transplant

• progression-free survival (PFS)

  From enrollment to 60 days post-transplant

• Adverse events

  From medication to 30 days after discontinuation

Study Arms (1)

Experimental group

EXPERIMENTAL

Drug: Hetrombopag

Interventions

Hetrombopag DRUG

Patients enrolled were administered Hetrombopag starting from +4d after transplantation on routine medication of recombinant human thrombopoietin injection (rhTPO). The starting dose of Hetrombopag is 2.5mg or 5mg, oral, once daily (starting at 5mg/d for body weight\>20kg; starting from 2.5mg/d for body weight ≤ 20kg). Adjust the dosage based on platelet count every two weeks, with a maximum dose of 7.5mg per day. Stop the medication after 6 weeks of continuous use or when PLT reaches 100 × 10\^9/L.

Experimental group

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age\<18 years old;
• Diagnosed as neuroblastoma;
• ECOG ≤ 2;
• First time receiving autologous hematopoietic stem cell transplantation;
• The subjects or their legal guardians agree to participate and sign the informed consent form.

You may not qualify if:

• ALT/AST \> 3 ULN, or TBIL \> 1.5 ULN;
• Have experienced congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring medication treatment within one year prior to enrollment, or have experienced myocardial infarction or cerebral infarction within three months prior to enrollment;
• Suffering from thromboembolic diseases;

Sponsors & Collaborators

• Beijing Children's Hospital: lead

Study Sites (1)

Department of Stem Cell Transplantation, Beijing Children's Hospital, Capital Medical University

Beijing, China

RECRUITING

MeSH Terms

Conditions

Neuroblastoma

Interventions

hetrombopag

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Central Study Contacts

Dr. Yang

CONTACT

86-010-59617614; yangjundabby@outlook.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Beijing Children's Hospital

Study Record Dates

• First Submitted: June 20, 2024
• First Posted: February 11, 2025
• Study Start: June 30, 2024
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): July 31, 2027
• Last Updated: December 3, 2025

Record last verified: 2025-11

Locations

CN (1)