Back to resultsPrevention of Severe Acute Graft-versus-host Disease in Pediatric Patients Using a daGOAT Model
Primary Purpose
Transplant-Related Disorder
Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Ruxolitinib
Sponsored by
About this trial
This is an interventional prevention trial for Transplant-Related Disorder
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Sites / Locations
- Institute of Hematology & Blood Diseases HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
The group of daGOAT model prevention
Arm 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.
Outcomes
Primary Outcome Measures
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).
Secondary Outcome Measures
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).
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.
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.
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.
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.
Total cost of treatment during 1.5 year after transplantation
Data on total cost of treatment will be collected from the medical records.
Full Information
NCT ID
NCT05599256
First Posted
October 20, 2022
Last Updated
March 22, 2023
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
1. Study Identification
Unique Protocol Identification Number
NCT05599256
Brief Title
Prevention of Severe Acute Graft-versus-host Disease in Pediatric Patients Using a daGOAT Model
Official Title
A Prospective, Single-arm Clinical Trial of Prevention of Severe Acute Graft-versus-host Disease After Pediatric Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation Using a daGOAT Model
Study Type
Interventional
2. Study Status
Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 9, 2023 (Actual)
Primary Completion Date
June 1, 2024 (Anticipated)
Study Completion Date
December 1, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Institute of Hematology & Blood Diseases Hospital, China
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transplant-Related Disorder
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
The group of daGOAT model prevention
Arm Type
Experimental
Arm 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.
Intervention Type
Drug
Intervention Name(s)
Ruxolitinib
Intervention 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.
Primary Outcome Measure Information:
Title
Severe aGVHD during 100 days after transplantation according to the MAGIC criteria
Description
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).
Time Frame
100 days after transplantation
Secondary Outcome Measure Information:
Title
aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria
Description
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).
Time Frame
100 days after transplantation
Title
Overall survival during 1.5 year after transplantation
Description
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.
Time Frame
Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation
Title
Relapse-free survival rate and relapse rate during 1.5 year after transplantation
Description
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.
Time Frame
Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation
Title
Incidence of infections during 1.5 year after transplantation
Description
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.
Time Frame
1.5 year after transplantation
Title
Safety of treatment during 100 days after transplantation according to the Common Terminology Criteria for Adverse Events version 5.0
Description
Data on adverse events of treatment will be collected.
Time Frame
100 days after transplantation
Title
Total cost of treatment during 1.5 year after transplantation
Description
Data on total cost of treatment will be collected from the medical records.
Time Frame
1.5 year after transplantation
10. Eligibility
Sex
All
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xueou Liu, PhD
Phone
022-23909051
Email
liuxueou@ihcams.ac.cn
Facility Information:
Facility Name
Institute of Hematology & Blood Diseases Hospital
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xueou Liu, PHD
Phone
022-23909051
Email
liuxueou@ihcams.ac.cn
12. IPD Sharing Statement
Learn more about this trial
Prevention of Severe Acute Graft-versus-host Disease in Pediatric Patients Using a daGOAT Model
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