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

A Prospective, Single-arm Clinical Trial of Prevention of Severe Acute Graft-versus-host Disease After Adult Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation Using a daGOAT Model

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.

Study Overview

• Status: Completed
• Conditions: Transplant-Related Disorder
• Intervention / Treatment: Drug: Ruxolitinib

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 Type: Interventional
• Enrollment (Actual): 115
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300020
      • Institute of Hematology & Blood Diseases Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: The group of daGOAT model prevention; 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; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria	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)	100 days after transplantation
Overall survival during 1.5 year 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.	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	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.	Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation
Incidence of infections during 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.	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	Data on adverse events of treatment will be collected.	100 days after transplantation
Total cost of treatment during 1.5 year after transplantation	Data on total cost of treatment will be collected from the medical records.	1.5 year after transplantation

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): January 15, 2023
• Primary Completion (Actual): October 10, 2024
• Study Completion (Actual): December 1, 2024

Study Registration Dates

• First Submitted: October 18, 2022
• First Submitted That Met QC Criteria: October 25, 2022
• First Posted (Actual): November 1, 2022

Study Record Updates

• Last Update Posted (Estimated): December 6, 2024
• Last Update Submitted That Met QC Criteria: December 4, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: IIT2022034（adult）

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No