Prevention of Severe Acute Graft-versus-host Disease in Pediatric Patients Using a daGOAT Model

Phase 2

Recruiting

• Conditions: Transplant-Related Disorder
• Interventions: Drug: Ruxolitinib

• Registration Number: NCT05599256
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Brief Summary

To evaluate the efficacy and safety of ruxolitinib for prophylactic therapy of child patients who are predicted to have a high risk for developing severe acute graftversus-host disease (aGVHD) by the dynamic aGVHD Onset Anticipation Tianjin (daGOAT) model.

Detailed Description

This study aims to prospectively evaluate the use of the daGOAT model in real-world clinical settings at the Institute of Hematology, Chinese Academy of Medical Sciences (IHCAMS).

Study Timeline

• Study First Submitted: 2022-10-20
• Study First Submitted QC: 2022-10-25
• Study First Posted: 2022-10-31
• Study Start: 2023-02-09
• Status Verified: 2024-03
• Primary Completion: 2024-10-07
• Last Update Submitted: 2024-11-04
• Last Update Posted: 2024-11-06
• Study Completion: 2025-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

1. Patients must be ≤ 16 years of age;
2. Patients receiving human leukocyte antigen mismatched and non-cord blood allogeneic hematopoietic stem cell transplantation;
3. Patients who can take oral medication;
4. Patients or their guardians have to sign an informed consent form before the start of the research procedure.

Exclusion Criteria

1. Tandem transplantation or multiple transplantations;
2. Patients who are allergic to or cannot tolerate ruxolitinib;
3. Mental or other medical conditions that make the patients unable to comply with the research treatment and monitoring requirements;
4. Patients who are pregnant or cannot take appropriate contraceptive measures during treatment;
5. Patients who are ineligible for the study due to other factors, or will bear great risk if participating in the study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
The group of daGOAT model prevention	Ruxolitinib	Model-predicted high-risk patients: weight ≤ 25 kg, ruxolitinib, 2.5mg bid po until at least day 60 post-transplant and terminated after day 100; weight \> 25 kg, ruxolitinib, 5mg bid po until at least day 60 post-transplant and terminated after day 100. If 'azoles' are taken concomitantly, ruxolitinib will start at half dose. If the patient tolerates ruxolitinib, the dose can be increased to 10mg bid po. Model-predicted low risk: regular aGVHD prophylactic regimens.

Primary Outcome Measures

Name	Time	Method
Severe aGVHD during 100 days after transplantation according to the MAGIC criteria	100 days after transplantation	Incidence of severe aGVHD after transplantation within 100 days. The medical records for each case wil be reviewed by two or three physicians to confirm the aGVHD diagnosis and grading (according to the MAGIC criteria).

Secondary Outcome Measures

Name	Time	Method
aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria	100 days after transplantation	Incidence of aGVHD (any grade) in various target organs. The medical records for each case wil be reviewed by two or three physicians to confirm the aGVHD diagnosis and grading (according to the MAGIC criteria).
Relapse-free survival rate and relapse rate during 1.5 year after transplantation	Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation	Patients will be followed up at days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation; data on relapse will be collected.
Incidence of infections during 1.5 year after transplantation	1.5 year after transplantation	Infection was defined as meeting one of the following criteria: culture-confirmed presence of bacteria or fungi in a sample collected from a sterile site; polymerase chain reaction-confirmed viremia at ≥ 5000 copies/ml for the cytomegalovirus or ≥ 10000 copies/ml for the Epstein-Barr virus; or body temperature ≥ 38 ℃ with culture-confirmed presence of pathogens from a non-sterile site.
Total cost of treatment during 1.5 year after transplantation	1.5 year after transplantation	Data on total cost of treatment will be collected from the medical records.
Overall survival during 1.5 year after transplantation	Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation	Patients will be followed up at days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation; data on survival will be collected.
Safety of treatment during 100 days after transplantation according to the Common Terminology Criteria for Adverse Events version 5.0	100 days after transplantation	Data on adverse events of treatment will be collected.

Trial Locations

Locations (1)

Institute of Hematology & Blood Diseases Hospital; 🇨🇳 Tianjin, Tianjin, China