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

Institute of Hematology & Blood Diseases Hospital, China

Status and phase: Enrolling; Phase 2

Conditions: Transplant-Related Disorder

Treatments: Drug: Ruxolitinib

Study type: Interventional

Funder types: Other

Identifiers

NCT05599256

IIT2022034（child）

Details and patient eligibility

About

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.

Full 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).

Enrollment: 40 estimated patients

Sex: All

Ages: Under 16 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

The group of daGOAT model prevention

Experimental group

Description:

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.

Treatment:

Drug: Ruxolitinib