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

NCT05599256 | Recruiting Phase 2

• 1 other identifier: IIT2022034（child）
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Transplant-Related Disorder

Outcome Measures

Primary Outcomes (1)

• Severe aGVHD during 100 days after transplantation according to the MAGIC criteria

  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).

  100 days after transplantation

Secondary Outcomes (6)

• aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria

  100 days after transplantation

• Overall survival during 1.5 year after transplantation

  Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation

• 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

• Incidence of infections during 1.5 year after transplantation

  1.5 year after transplantation

• Safety of treatment during 100 days after transplantation according to the Common Terminology Criteria for Adverse Events version 5.0

  100 days after transplantation

Study Arms (1)

The group of daGOAT model prevention

EXPERIMENTAL

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.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

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.

The group of daGOAT model prevention

Eligibility Criteria

• Age: Up to 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

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

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Interventions

ruxolitinib

Central Study Contacts

Xueou Liu, PhD

CONTACT

022-23909051; liuxueou@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 20, 2022
• First Posted: October 31, 2022
• Study Start: February 9, 2023
• Primary Completion: October 7, 2024
• Study Completion: December 1, 2025
• Last Updated: November 6, 2024

Record last verified: 2024-03

Locations

CN (1)