Acute Graft Versus Host DiseasePhase 3NCT05921305
Mini-dose MTX Plus Standard-dose Steroid for the Initial Treatment of Acute GVHD
A Phase 3 study testing MTX and Corticosteroid in people with Acute Graft Versus Host Disease, run by Peking University People's Hospital at 2 sites in China.
Trial summary
This trial is a randomized (1:1) phase III open label study of frontline mini-MTX plus methylprednisolone 2mg/kg/day compared to methylprednisolone 2mg/kg in allogeneic stem cell transplant recipients with grade 2-4 aGVHD. Read more about this study
- Who is it for?
- Ages 15 to 65 years
- Conditions the study is for
- Acute Graft Versus Host Disease
- What is being tested?
- MTX and Corticosteroid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 218 participants
- Where
- 2 sitesChina
- Study dates
- 3 Aug 2023to 30 Sep 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 15 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients who are fully informed and sign informed consent by themselves or their guardians;
- Patients receiving first allogeneic hematopoietic stem cell transplantation;
- Patients with acute graft-versus-host disease of grade II-IV were diagnosed after transplantation;
- KPS>60, Estimated survival >3 months;
- No serious organ damage:
- ANC in peripheral blood is greater than 0.5×109/l
- Creatinine < 1.5mg/dl
- Cardiac ejection index > 55%
Who cannot join
- Patients with severe brain, heart, kidney or liver dysfunction unrelated to graft-versus host disease;
- Patients with uncontrollable active infection;
- Patients with recurrence of primary malignant hematopathy;
- Expected survival is less than 3 months
- Patients who have histories of severe allergic reactions
- Pregnant or lactating women
- The researcher judges that there are other factors that are not suitable for participating
- Patients who received donor lymphocyte infusion
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Xiao-Jun Huang, M.D. contact
China2 sites, 2 recruiting
- Recruiting
Peking University Institute of Hematology,
Beijing, Beijing Municipality, 100044
Yu Wang, Dr., 13552647384, ywyw3172@sina.com
- Recruiting
Nanfang Hospital, Nanfang Medical University
Guangzhou, Guangdong, 510515
qifa liu, M.D., liuqifa628@163.com
qifa liu, M.D.
About this study
Official title: Randomized Trial of Mini-dose Methotrexate Plus Standard-dose Steroid vs Steroids for the Initial Treatment of Acute Graft Versus Host Disease
This trial is a randomized (1:1) phase III open label study of frontline mini-MTX plus methylprednisolone 2mg/kg/day compared to methylprednisolone 2mg/kg in allogeneic stem cell transplant recipients with grade 2-4 aGVHD.
Read the detailed description
Allo-HSCT is an effective treatment of malignant hematopoietic diseases. However, aGVHD remains a major complication after allo-HSCT. Corticosteroid is the standard first-line therapy for aGVHD. However, the response rate of corticosteroid was approximate 50%, and the clinical outcomes of patients with corticosteroid refractory GVHD were poor. Thus far, no combination therapy had been prove to be superior to corticosteroid alone as initial therapy for aGVHD. The study hypothesis: MTX combined corticosteroid treatment could help to control aGVHD.
- Conditions
- Acute Graft Versus Host Disease
- Being tested
- MTX, Corticosteroid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MTXDrug
MTX (5mg/m^2/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR
CorticosteroidDrug
Corticosteroid Methylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone).
| Study group | Role | What they receive |
|---|---|---|
| MTX and corticosteroid | Experimental | Methylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). MTX (5mg/m^2/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR. Receives: Drug: MTX |
| Corticosteroid | Active comparator | Methylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). Receives: Drug: Corticosteroid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR) for aGVHD treatment after treatment
Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response
Measured: 14 days
Secondary outcomes (9)
Overall response rate (ORR) for aGVHD treatment at 28 days after treatment
Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response
Measured: 28 days
Overall response rate (ORR) for aGVHD treatment at 42 days after treatment
Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response
Measured: 42 days
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Data collection including questionnaires at individual and group visits and physician interviews at individual visits will be used to assess participants for treatment-related adverse events.
Measured: 42 days
cGVHD
The cumulative incidence of chronic GVHD
Measured: 1 years
Infection and poor graft function
The cumulative incidence of severe infection and poor graft function
Measured: 1 year
Relapse
The cumulative incidence of relapse
Measured: 1 year
Non-relapse mortality
The cumulative incidence of non-relapse mortality
Measured: 1 year
Overall survival
The cumulative incidence of overall survival
Measured: 1 year
Disease free survival
The cumulative incidence of disease free survival
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2023
- Study start3 Aug 2023
- Primary completion30 Jun 2026 (estimated)
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Graft Versus Host DiseaseStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 218 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05921305
- Sponsor's protocol ID
- GVHD-MTX-Randomized trial
- Status last verified
- May 2025
- First submitted
- 19 Jun 2023
- Last updated
- 3 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Trial of Mini-dose Methotrexate Plus Standard-dose Steroid vs Steroids for the Initial Treatment of Acute Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT05921305
- Acronym / sponsor ID
- GVHD-MTX-Randomized trial
- Conditions
- Acute Graft Versus Host Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 218 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 3 Aug 2023 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 27 Jun 2023 (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.
This trial is a randomized (1:1) phase III open label study of frontline mini-MTX plus methylprednisolone 2mg/kg/day compared to methylprednisolone 2mg/kg in allogeneic stem cell transplant recipients with grade 2-4 aGVHD.
Detailed description
Allo-HSCT is an effective treatment of malignant hematopoietic diseases. However, aGVHD remains a major complication after allo-HSCT. Corticosteroid is the standard first-line therapy for aGVHD. However, the response rate of corticosteroid was approximate 50%, and the clinical outcomes of patients with corticosteroid refractory GVHD were poor. Thus far, no combination therapy had been prove to be superior to corticosteroid alone as initial therapy for aGVHD. The study hypothesis: MTX combined corticosteroid treatment could help to control aGVHD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MTX and corticosteroid | Experimental | Drug: MTXMethylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). MTX (5mg/m^2/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR. |
| Corticosteroid | Active comparator | Drug: CorticosteroidMethylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). |
Who may qualify
- Ages
- 15 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Patients who are fully informed and sign informed consent by themselves or their guardians;
- Patients receiving first allogeneic hematopoietic stem cell transplantation;
- Patients with acute graft-versus-host disease of grade II-IV were diagnosed after transplantation;
- KPS>60, Estimated survival >3 months;
- No serious organ damage:
Key exclusion criteria (8 listed)
- Patients with severe brain, heart, kidney or liver dysfunction unrelated to graft-versus host disease;
- Patients with uncontrollable active infection;
- Patients with recurrence of primary malignant hematopathy;
- Expected survival is less than 3 months
- Patients who have histories of severe allergic reactions
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 |
|---|---|---|
| MTXMTX (5mg/m^2/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR | Drug | MTX and corticosteroid |
| CorticosteroidCorticosteroid Methylprednisolone 2 mg/kg/day was given for 3 days, and for responding patients, the dose of prednisone must be at least 0.25mg/kg/day prednisone (or 0.2mg/kg/day methylprednisolone). | Drug | Corticosteroid |
Primary outcome
Overall response rate (ORR) for aGVHD treatment after treatment
Time frame: 14 days
Overall response rate is defined as the proportion of patients demonstrating a complete response or partial response without requirement for additional systemic therapies for an earlier progression, mixed response or non-response
9 secondary outcomes
Overall response rate (ORR) for aGVHD treatment at 28 days after treatment
Time frame: 28 days
Overall response rate (ORR) for aGVHD treatment at 42 days after treatment
Time frame: 42 days
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 42 days
cGVHD
Time frame: 1 years
Infection and poor graft function
Time frame: 1 year
Relapse
Time frame: 1 year
Non-relapse mortality
Time frame: 1 year
Overall survival
Time frame: 1 year
Disease free survival
Time frame: 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2025.
- Individual participant dataSharing planned: No
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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.
Xiao-Jun Huang, M.D. contact
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MTX and Corticosteroid in people with Acute Graft Versus Host Disease, run by Peking University People's Hospital at 2 sites 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 8 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 3 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yu Wang, M.D. (86-13552647384, ywyw3172@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (ORR) for aGVHD treatment after treatment (time frame: 14 days). 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 3 Jun 2025; AzTrials last refreshed its copy on 28 Oct 2026.