Hetrombopag for the Enhancement of Platelet Engraftment After Allogeneic Hematopoietic Stem Cell Transplantation

NCT05727917 | Unknown

• 1 other identifier: 20230131
• Study Type: interventional
• Target: 78
• Locations: 1 country
• Sites: 1

Brief Summary

This investigator-initiated, prospective, multicenter, open-label, randomized, controlled clinical study is designed to evaluate the clinical efficacy and safety of hetrombopag for promoting platelet engraftment after allo-HSCT in patients with hematological disease. After signing the informed consent form, the patients will enter the screening period (up to 14 days), and the qualified patients will be randomly selected into the experimental group and the control group according to the ratio of 1:1. Experimental group: After hematopoietic stem cell reinfusion, the patients begin to take hetrombopag orally 7.5mg/d, until the patients reach complete platelet response (CR, platelet count ≥ 50×109/L for 3 consecutive days without platelet transfusions for 7 consecutive days). The treatment will stop when patients accept 21 consecutive days of treatment or reach the discontinuation criteria. Control group: After hematopoietic stem cell transfusion, the patients will be only observed, and the observation during the treatment period will be ended after 30 days. Patients will continue to enter the follow-up period (+ 100 days after transplantation) and the survival follow-up period (1 year after transplantation) after the end of the treatment period.

Conditions

Hematological Disease; Allogeneic Hematopoietic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• Proportion of patients with platelet engraftment within 21 days after transplantation

  Proportion of patients who maintained platelet count ≥ 20 × 10\^9/L without platelet transfusion for 7 consecutive days within 21 days after transplantation

  21 days after transplantation

Secondary Outcomes (6)

• Median time to platelet engraftment

  100 days

• Median time to neutrophil engraftment

  100 days

• Proportion of patients with neutrophil engraftment within 21 days after transplantation

  21 days after transplantation

• Proportion of patients with platelet engraftment within 30 days after transplantation

  30 days after transplantation

• Platelet complete response rate (CR) within 30 days after transplantation

  30 days after transplantation

Study Arms (2)

hetrombopag group (Experimental group)

EXPERIMENTAL

After hematopoietic stem cell reinfusion, the patients begin to take hetrombopag orally 7.5mg/d, until the patients reach complete platelet response (CR, platelet count ≥50×109/L for 3 consecutive days without platelet transfusions for 7 consecutive days). The treatment will stop when patients accept 21 consecutive days of treatment or reach the discontinuation criteria.

Drug: Hetrombopag

Control group

PLACEBO COMPARATOR

After hematopoietic stem cell transfusion, the patients will be only observed, and the observation during the treatment period will be ended after 30 days.

Other: placebo

Interventions

Hetrombopag DRUG

After hematopoietic stem cell reinfusion, the patients begin to take hetrombopag orally 7.5mg/d, until the patients reach complete platelet response.The treatment will stop when patients accept 21 consecutive days of treatment or reach the discontinuation criteria.

hetrombopag group (Experimental group)

placebo OTHER

After hematopoietic stem cell transfusion, the patients will be only observed, and the observation during the treatment period will be ended after 30 days

Control group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• The patients signed the informed consent and participated in the study voluntarily;
• Age ≥ 18 years old, regardless of gender;
• ECOG score ≤ 2;
• Expected survival ≥ 3 months;
• Patients with hematological diseases who received allo-HSCT for the first time;
• The patient's laboratory tests met the following criteria:
• Alanine aminotransferase (ALT) ≤ 3.0 × upper limit of normal (ULN), Aspartate aminotransferase (AST) ≤ 3.0 × ULN;
• Serum total bilirubin ≤ 1.5 × ULN;
• Serum creatinine ≤ 1.5 × ULN;
• Investigators evaluated that patients would benefit from hetrombopag.

You may not qualify if:

• History of allergy to TPO-RA drugs;
• Previous bone marrow or hematopoietic stem cell transplantation;
• Hematologic malignancies did not reach complete remission before transplantation;
• Central nervous system leukemia;
• Arterial or venous thrombosis, such as cerebral infarction, pulmonary embolism, arterial thrombosis, deep venous thrombosis and disseminated intravascular coagulation (DIC), occurred within 6 months before the screening period;
• Heart disease, such as New York Heart Association (NYHA) class III/IV congestive heart failure, angina pectoris, myocardial infarction, and arrhythmias requiring treatment, occurring within 6 months before the screening period;
• Prolonged QT syndrome during the screening period;
• Active hepatitis B or hepatitis C;
• Positive for anti-HIV antibodies or anti-TP antibodies;
• Active infection that is difficult to control;
• Pregnant or breastfeeding women;

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300041, China

RECRUITING

MeSH Terms

Conditions

Hematologic Diseases

Interventions

hetrombopag

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases

Study Officials

• Erlie Jiang

  Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Aiming Pang

CONTACT

+86-13820398091; pangaiming@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2023
• First Posted: February 14, 2023
• Study Start: January 1, 2023
• Primary Completion: May 31, 2024
• Study Completion: October 31, 2024
• Last Updated: February 14, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)