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

NCT05600855 | Completed Phase 2

• 1 other identifier: IIT2022034（adult）
• Study Type: interventional
• Target: 115
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the efficacy and safety of ruxolitinib for prophylactic therapy of adult 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: ruxolitinib 5mg bid po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (\<0.1×10\^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery. Model-predicted moderate-risk patients: ruxolitinib 2.5mg bid p po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (\<0.1×10\^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery. Model-predicted low risk: regular aGVHD prophylactic regimens.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Model-predicted high-risk patients: ruxolitinib 5mg bid po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (\<0.1×10\^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery. Model-predicted moderate-risk patients: ruxolitinib 2.5mg bid p po until at least day 60 post-transplant and terminated after day 100. If severe hematological signs occur such as when there is severe neutropenia (\<0.1×10\^9/L), ruxolitinib can be used at half dose or discontinued as appropriate, and can continue to be used after hematology recovery. Model-predicted low risk: regular aGVHD prophylactic regimens.

The group of daGOAT model prevention

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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 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

Location

Related Publications (1)

• Chen J, Cao Y, Feng Y, Qi S, Yang D, Hu Y, Pang A, Shen Q, Luo J, Gong X, Zhang R, Zhai X, Li X, Yan W, Zhang X, Chen M, Niu M, Wei J, Liang C, Zhai W, Zhao N, Liu X, Liu S, Zhai W, Li R, Shao X, Zhang D, Wang M, Pan P, Xu M, Zhang W, Xu Y, Zhu X, Guo Y, Wang H, Song Z, Gale RP, Han M, Feng S, Jiang E. Autonomous artificial intelligence prescribing a drug to prevent severe acute graft-versus-host disease in HLA-haploidentical transplants. Nat Commun. 2025 Sep 25;16(1):8391. doi: 10.1038/s41467-025-62926-0.

  PMID: 40998766 DERIVED

MeSH Terms

Interventions

ruxolitinib

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 18, 2022
• First Posted: November 1, 2022
• Study Start: January 15, 2023
• Primary Completion: October 10, 2024
• Study Completion: December 1, 2024
• Last Updated: December 6, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)