Transplant-Related DisorderPhase 2NCT05600855
Prevention of Severe Acute Graft-versus-host Disease in Adult Patients Using a daGOAT Model
A Phase 2 study testing Ruxolitinib in people with Transplant-Related Disorder, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Transplant-Related Disorder
- What is being tested?
- Ruxolitinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 115 participants
- Where
- 1 siteChina
- Study dates
- 15 Jan 2023to 1 Dec 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
About this study
Official title: 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.
Read the 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).
- Conditions
- Transplant-Related Disorder
- Being tested
- Ruxolitinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ruxolitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 100 days after transplantation
Secondary outcomes (6)
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)
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 1.5 year after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2022
- Study start15 Jan 2023
- Primary completion10 Oct 2024
- Study completion1 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Transplant-Related DisorderStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 115 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05600855
- Sponsor's protocol ID
- IIT2022034（adult）
- Status last verified
- Dec 2024
- First submitted
- 18 Oct 2022
- Last updated
- 6 Dec 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Publications
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.
PubMed 40998766
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05600855
- Acronym / sponsor ID
- IIT2022034（adult）
- Conditions
- Transplant-Related Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 115 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 15 Jan 2023 (actual)
- Primary completion
- 10 Oct 2024 (actual)
- Study completion
- 1 Dec 2024 (actual)
- First posted
- 1 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| The group of daGOAT model prevention | Experimental | Drug: RuxolitinibModel-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. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RuxolitinibModel-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 | The group of daGOAT model prevention |
Primary outcome
Severe aGVHD during 100 days after transplantation according to the MAGIC criteria
Time frame: 100 days after transplantation
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)
6 secondary outcomes
aGVHD in various target organs during 100 days after transplantation according to the MAGIC criteria
Time frame: 100 days after transplantation
Overall survival during 1.5 year after transplantation
Time frame: 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
Time frame: Days 14, 28, 42, 60, 90, 180, 270, 360 and 540 after transplantation
Incidence of infections during 1.5 year after transplantation
Time frame: 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
Time frame: 100 days after transplantation
Total cost of treatment during 1.5 year after transplantation
Time frame: 1.5 year after transplantation
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2024.
- Derived from this study: PubMed 40998766Chen 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.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib in people with Transplant-Related Disorder, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Severe aGVHD during 100 days after transplantation according to the MAGIC criteria (time frame: 100 days after transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05600855, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Dec 2024.