Transplant-Related DisorderPhase 2NCT05599256
Prevention of Severe Acute Graft-versus-host Disease in Pediatric 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 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. Read more about this study
- Who is it for?
- Ages Up to 16 years
- 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
- 40 participants
- Where
- 1 siteChina
- Study dates
- 9 Feb 2023to 1 Dec 2025
- 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 Up to 16 years
- All sexes
- No healthy volunteers
- Child
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 or their guardians 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, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Xueou Liu, PHD, 022-23909051, liuxueou@ihcams.ac.cn
About this study
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
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.
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: 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. 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 posted31 Oct 2022
- Study start9 Feb 2023
- Primary completion7 Oct 2024
- Study completion1 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 40 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05599256
- Sponsor's protocol ID
- IIT2022034（child）
- Status last verified
- Mar 2024
- First submitted
- 20 Oct 2022
- Last updated
- 6 Nov 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 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 Pediatric Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation Using a daGOAT Model
- ClinicalTrials.gov ID
- NCT05599256
- Acronym / sponsor ID
- IIT2022034（child）
- Conditions
- Transplant-Related Disorder
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 9 Feb 2023 (actual)
- Primary completion
- 7 Oct 2024 (actual)
- Study completion
- 1 Dec 2025 (estimated)
- First posted
- 31 Oct 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 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).
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: 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. |
Who may qualify
- Ages
- Up to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 or their guardians 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: 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 | 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
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2024.
Related trials for Transplant-Related Disorder
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingObservational study
Preoperative Gastric Ultrasound and Left Ventricular Function in Coronary Artery Bypass Surgery
Coronary Artery Disease, Left Ventricular Systolic Dysfunction
Active, not recruitingObservational study
Retinal Pigment Epithelium Safety Study For Patients In B4711001
Age Related Macular Degeneration
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
Active, not recruitingPhase 2
Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency
DOCK8 Deficiency
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.
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: status not confirmed, as of the sponsor's last update on 6 Nov 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xueou Liu, PhD (022-23909051, liuxueou@ihcams.ac.cn), or a site near the patient. 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?
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 Nov 2024.