Acute GVH DiseasePhase 3NCT04960644
MTX and Steroid as First-line Therapy for aGVHD
A Phase 3 study testing MTX and Corticosteroid in people with Acute GVH Disease, run by Peking University People's Hospital at 1 site in China.
Trial summary
The aim of the study is to identify the efficacy and safety of methotrexate (MTX) combined corticosteroid as first-line therapy for acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (alloHSCT). Read more about this study
- Who is it for?
- Ages 15 to 65 years
- Conditions the study is for
- Acute GVH Disease
- What is being tested?
- MTX and Corticosteroid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 157 participants
- Where
- 1 siteChina
- Study dates
- 25 Jun 2021to 31 Dec 2022
- Phase
- Phase 3
- 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 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 allogeneic hematopoietic stem cell transplantation;
- Patients with acute graft-versus-host disease of grade II-IV were diagnosed after transplantation;
- Patients with stable implantation of granulocytes and platelets.
- ECOG score ≤3
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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Multi-cohort, Phase Ⅲ Study of Methotrexate Combined Corticosteroid as First-line Therapy in Chinese Patients With Acute Graft vs. Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
The aim of the study is to identify the efficacy and safety of methotrexate (MTX) combined corticosteroid as first-line therapy for acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (alloHSCT).
Read the detailed description
Allo-HSCT is an effective treatment of malignant hematopoietic diseases. However, aGVHD remains a major complication after allo-HSCT and the destruction of recipient tissues by alloantigen-activated T cells is a key event in the development of aGVHD. Corticosteroid is the standard first-line therapy for aGVHD due to their roles in suppressing T cell responses. 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 further ameliorate the activity of T cells and control aGVHD.
- Conditions
- Acute GVH Disease
- Being tested
- MTX, Corticosteroid
- Keywords
- acute GVHD, MTX, Corticosteroid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MTXDrug
Also called Corticosteroid
MTX (5-10 mg/day,Dmax 10mg/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR
CorticosteroidDrug
corticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then the dosage was gradually reduced according to patient's response
| Study group | Role | What they receive |
|---|---|---|
| MTX and corticosteroid | Experimental | perimental: MTX and corticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then gradually reduce the dose according to patient's response MTX (5-6mg/m^2/day,Maximum dose 10mg/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR Receives: Drug: MTX |
| corticosteroid | Active comparator | perimental: corticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then gradually reduce the dose according to patient's response 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: 10 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 posted14 Jul 2021
- Study start25 Jun 2021
- Primary completion31 Jul 2022
- Study completion31 Dec 2022
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 Acute GVH DiseaseStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 157 participants (actual)
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
- Investigators
- Xiao-Jun Huang, Dr., Principal investigator, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04960644
- Sponsor's protocol ID
- NFEC-202105-K3-01
- Status last verified
- May 2025
- First submitted
- 23 Jun 2021
- Last updated
- 3 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Wang Y, Liu QF, Wu DP, Xu ZL, Han TT, Sun YQ, Huang F, Fan ZP, Xu N, Chen F, Zhao Y, Kong Y, Mo XD, Xu LP, Zhang XH, Liu KY, Huang XJ. Mini-dose methotrexate combined with methylprednisolone for the initial treatment of acute GVHD: a multicentre, randomized trial. BMC Med. 2024 Apr 25;22(1):176. doi: 10.1186/s12916-024-03395-y.
PubMed 38664766
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
- A Multi-cohort, Phase Ⅲ Study of Methotrexate Combined Corticosteroid as First-line Therapy in Chinese Patients With Acute Graft vs. Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT04960644
- Acronym / sponsor ID
- NFEC-202105-K3-01
- Conditions
- Acute GVH Disease
- Keywords
- acute GVHD, MTX, Corticosteroid
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 157 participants (actual)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 25 Jun 2021 (actual)
- Primary completion
- 31 Jul 2022 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 14 Jul 2021 (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.
The aim of the study is to identify the efficacy and safety of methotrexate (MTX) combined corticosteroid as first-line therapy for acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (alloHSCT).
Detailed description
Allo-HSCT is an effective treatment of malignant hematopoietic diseases. However, aGVHD remains a major complication after allo-HSCT and the destruction of recipient tissues by alloantigen-activated T cells is a key event in the development of aGVHD. Corticosteroid is the standard first-line therapy for aGVHD due to their roles in suppressing T cell responses. 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 further ameliorate the activity of T cells and 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: MTXperimental: MTX and corticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then gradually reduce the dose according to patient's response MTX (5-6mg/m^2/day,Maximum dose 10mg/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR |
| corticosteroid | Active comparator | Drug: Corticosteroidperimental: corticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then gradually reduce the dose according to patient's response |
Who may qualify
- Ages
- 15 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Patients who are fully informed and sign informed consent by themselves or their guardians;
- Patients receiving allogeneic hematopoietic stem cell transplantation;
- Patients with acute graft-versus-host disease of grade II-IV were diagnosed after transplantation;
- Patients with stable implantation of granulocytes and platelets.
- ECOG score ≤3
Key exclusion criteria (7 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 |
|---|---|---|
| MTXAlso: CorticosteroidMTX (5-10 mg/day,Dmax 10mg/day) was given on days 1, 3, and 8, and repeated weekly until aGVHD was CR | Drug | MTX and corticosteroid |
| Corticosteroidcorticosteroid Methylprednisolone 1 mg/kg/day was given for 10 days and then the dosage was gradually reduced according to patient's response | Drug | corticosteroid |
Primary outcome
Overall response rate (ORR) for aGVHD treatment after treatment
Time frame: 10 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
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 3 Jun 2025.
- Derived from this study: PubMed 38664766Wang Y, Liu QF, Wu DP, Xu ZL, Han TT, Sun YQ, Huang F, Fan ZP, Xu N, Chen F, Zhao Y, Kong Y, Mo XD, Xu LP, Zhang XH, Liu KY, Huang XJ. Mini-dose methotrexate combined with methylprednisolone for the initial treatment of acute GVHD: a multicentre, randomized trial. BMC Med. 2024 Apr 25;22(1):176. doi: 10.1186/s12916-024-03395-y.
- 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. This study is not recruiting at the moment.
Xiao-Jun Huang, Dr. principal investigator
Peking University People's Hospital
1 site 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 GVH Disease, run by Peking University People's Hospital 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 5 inclusion and 7 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 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 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 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?
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?
Overall response rate (ORR) for aGVHD treatment after treatment (time frame: 10 days). 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 NCT04960644, 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 3 Jun 2025.