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GVHD, AcutePhase 2GVHDNCT07509749

Novel Therapies for Severe Acute GVHD (Graft-versus-host Disease)

A Phase 2 study testing Ruxolitinib and Methylprednisolone and Methylprednisolone in people with GVHD, Acute and Stem Cell Transplant Complications, run by Daihong Liu at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The purpose of this study is to determine the efficacy and safety of combined Ruxolitinib With Corticosteroids as First Line Therapy for the severe acute GVHD (graft-versus-host disease ) Read more about this study

Who is it for?
Ages 14 years and older
What is being tested?
Ruxolitinib and Methylprednisolone and Methylprednisolone
Could I receive a placebo?
No, everyone receives an active treatment
How many people
54 participants
Where
1 siteChina
Study dates
30 Jun 2025to 30 Dec 2027
Phase
Phase 2
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 14 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • diagnosed with hematological diseases.
  • Have undergone first allogeneic hematopoietic stem cell transplantation (allo-HSCT) from any donor source using bone marrow, peripheral blood stem cells, or cord blood for hematologic malignancies.
  • New onset of severe acute GVHD within 100 days post-transplantation.

Who cannot join

  • Recipients of second allogeneic stem cell transplant.
  • Acute GVHD induced by donor lymphocyte infusion, interferon.
  • Received first line aGVHD treatment before enrollment.
  • Overlap GVHD syndrome.
  • Pregnant or breast-feeding women.
  • Absolute neutrophil count (ANC) <0.5×10e9/L or platelet count (PLT) < 20×10e9/L.
Show all 17Show fewer
  • Serum creatinine > 2.0 mg/dL or creatinine clearance < 40 mL/min measured or calculated by Cockroft-Gault equation.
  • Uncontrolled infection.
  • Human immunodeficiency virus infection.
  • Active hepatitis b virus, hepatitis C virus infection and need antivirus treatment.
  • Subjects with evidence of relapsed primary disease, or subjects who have been treated for relapse after the allo-HSCT was performed, or graft rejection.
  • Allergic history to Janus kinase inhibitors.
  • Severe organ dysfunction unrelated to underlying GVHD, including:
  • (1)Cholestatic disorders or unresolved veno-occlusive disease of the liver (defined as persistent bilirubin abnormalities not attributable to GVHD and ongoing organ dysfunction).
  • (2)Clinically significant or uncontrolled cardiac disease including unstable angina, acute myocardial infarction within 6 months from Day 1 of study drug administration, New York Heart Association Class III or IV congestive heart failure, circulatory collapse requiring vasopressor or inotropic support, or arrhythmia that requires therapy.
  • (3)Clinically significant respiratory disease that requires mechanical ventilation support or 50% oxygen.
  • 14.Received Janus kinase inhibitor therapy after allo-HSCT for any indication. 15.Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits; pose a significant risk to the subject; or interfere with interpretation of study data.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Daihong Liu contact

01066937079

daihongrm@163.com

China1 site, 1 recruiting

  • Department of Hematology, the Fifth Center of Chinese PLA General Hospital

    Beijing, 100853

    Daihong Liu, +861066937079, daihongrm@163.com

    Daihong Liu, daihongrm@163.com

    Recruiting

Other trials in China

About this study

Official title: Prospective Randomized Controlled Trial of Novel Therapies for Severe Acute GVHD (Graft-versus-host Disease)

The purpose of this study is to determine the efficacy and safety of combined Ruxolitinib With Corticosteroids as First Line Therapy for the severe acute GVHD (graft-versus-host disease )

Read the detailed description

Acute graft-versus-host disease (GVHD) is treated with systemic corticosteroid immunosuppression as first line therapy. Many patients with severe acute GVHD do not respond to primary therapy, high-dose systemic corticosteroids; therefore, survival for those patients remains particularly poor. Here we determine the efficacy and safety of combined Ruxolitinib With Corticosteroids as First Line Therapy for the Treatment of severe acute GVHD.

Being tested
Ruxolitinib and Methylprednisolone, Methylprednisolone
Keywords
acute graft-versus-host disease, Ruxolitinib, first line therapy
Medical subject headings
Graft vs Host Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ruxolitinib and MethylprednisoloneDrug

    Participants began oral administration of ruxolitinib at 5 mg QD; Methylprednisolone (1mg/kg)

  • MethylprednisoloneDrug

    Methylprednisolone 2mg/kg/d , iv or iv gtt for at least 1 week, then taper according to the clinical response.

Study groups in NCT07509749
Study groupRoleWhat they receive
Ruxolitinib ArmExperimentalRuxolitinib(5mg/d) combined with Methylprednisolone(1mg/kg)
Receives: Drug: Ruxolitinib and Methylprednisolone; Drug: Methylprednisolone
Comparator armActive comparatorMethylprednisolone (2mg/kg)
Receives: Drug: Methylprednisolone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall response rate (ORR) at Day 28

    Defined as the proportion of participants demonstrating a complete response (CR), and partial response (PR).

    Measured: Day 28 after treatment

Secondary outcomes (8)

  1. Six-month duration of response

    Defined as the time from first response until graft-versus-host disease (GVHD) progression or death. Six-month duration of response will be assessed when all participants who are still on study complete the Day 180 visit.

    Measured: Six-month after treatment

  2. Ninety-day duration of response

    Defined as the time from first response until GVHD progression or death, when all participants who are still on study complete the Day 90 visit.

    Measured: Day 90 after treatment

  3. Nonrelapse mortality (NRM)

    NRM was defined as death from any cause without relapse. Cumulative incidence of NRM was analyzed in a competing risk framework using Gray's method.

    Measured: 1 year after treatment

  4. Cumulative incidence of relapse

    Defined as the proportion of participants whose underlying malignancy relapsed.Relapse was defined as hematologic recurrence of malignancies after transplantation. Cumulative incidence of relapse was analyzed in a competing risk framework using Gray's method.

    Measured: 1 year after treatment

  5. Disease-free survival (DFS)

    Defined as the time from first dose of ruxolitinib to the earliest date that a participant died, had a relapse/progression of the underlying malignancy, required additional therapy for aGVHD, or demonstrated signs or symptoms of chronic graft-versus-host disease (cGVHD).DFS will be evaluated in an intent-to-treat analysis by Kaplan Meier estimate and Log Rank test. Survival will be calculated from the date of randomization.

    Measured: 1 year after treatment

  6. GVHD-free and relapse-free survival (GRFS)

    GRFS was defined as the time onset of grade 3 to 4 aGVHD, moderate to severe cGVHD, or relapse/disease progression/death. GRFS will be evaluated in an intent-to-treat analysis by Kaplan Meier estimate and Log Rank test.

    Measured: 1 year after treatment

  7. recurrence of aGVHD

    Defined as the proportion of participants whose aGVHD relapsed.Relapse was defined as recurrence of new GVHD related symptoms after complete remission of aGVHD. Cumulative incidence of recurrence of aGVHD was analyzed in a competing risk framework using Gray's method.

    Measured: 1 year after treatment

  8. Failure-free survival

    Failure-free survival (FFS) refers to the time from randomization to disease relapse or progression, non-relapse mortality, or the addition of new therapy for aGVHD.

    Measured: 1 year after treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Apr 2026
  2. Study start30 Jun 2025
  3. Primary completion30 Dec 2027 (estimated)
  4. Study completion30 Dec 2027 (estimated)

Resources

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
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
54 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07509749
Sponsor's protocol ID
S2025-772-01
Acronym
GVHD
Status last verified
Mar 2026
First submitted
5 Jan 2026
Last updated
3 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: Data requests should be evaluated on a case-by-case basis by an independent review committee, in accordance with the moderated access policy of the Data Repository Unit at the Chinese PLA General Hospital, Beijing, China.

Publications

  • Betts BC, Bastian D, Iamsawat S, Nguyen H, Heinrichs JL, Wu Y, Daenthanasanmak A, Veerapathran A, O'Mahony A, Walton K, Reff J, Horna P, Sagatys EM, Lee MC, Singer J, Chang YJ, Liu C, Pidala J, Anasetti C, Yu XZ. Targeting JAK2 reduces GVHD and xenograft rejection through regulation of T cell differentiation. Proc Natl Acad Sci U S A. 2018 Feb 13;115(7):1582-1587. doi: 10.1073/pnas.1712452115. Epub 2018 Jan 30.

    PubMed 29382747

  • Zeiser R, von Bubnoff N, Butler J, Mohty M, Niederwieser D, Or R, Szer J, Wagner EM, Zuckerman T, Mahuzier B, Xu J, Wilke C, Gandhi KK, Socie G; REACH2 Trial Group. Ruxolitinib for Glucocorticoid-Refractory Acute Graft-versus-Host Disease. N Engl J Med. 2020 May 7;382(19):1800-1810. doi: 10.1056/NEJMoa1917635. Epub 2020 Apr 22.

    PubMed 32320566

  • Michonneau D, Devillier R, Keranen M, Rubio MT, Nicklasson M, Labussiere-Wallet H, Carre M, Huynh A, Viayna E, Roset M, Finzi J, Pfeiffer M, Thunstrom D, Lara N, Sabatelli L, Chevallier P, Itala-Remes M. Treatment Patterns and Clinical Outcomes of Patients with Moderate to Severe Acute Graft-Versus-Host Disease: A Multicenter Chart Review Study. Hematol Rep. 2024 May 6;16(2):283-294. doi: 10.3390/hematolrep16020028.

    PubMed 38804281

  • Mehta AK, Koreth J. Toward Improving Initial Therapy of Acute Graft Versus Host Disease. Am J Hematol. 2025 May;100 Suppl 3:40-54. doi: 10.1002/ajh.27593. Epub 2025 Feb 12.

    PubMed 39936555

  • Dou L, Zhao Y, Yang J, Deng L, Wang N, Zhang X, Liu Q, Yang Y, Wei Z, Wang F, Jiao Y, Li F, Luan S, Hu L, Gao S, Liu C, Liu X, Yan J, Zhang X, Zhou F, Lu P, Liu D. Ruxolitinib plus steroids for acute graft versus host disease: a multicenter, randomized, phase 3 trial. Signal Transduct Target Ther. 2024 Oct 23;9(1):288. doi: 10.1038/s41392-024-01987-x.

    PubMed 39438467

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib and Methylprednisolone and Methylprednisolone in people with GVHD, Acute and Stem Cell Transplant Complications, run by Daihong Liu 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 3 inclusion and 17 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 Apr 2026. 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?

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 Daihong Liu (01066937079, daihongrm@163.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) at Day 28 (time frame: Day 28 after treatment). 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 NCT07509749, which also lists 5 linked publications.

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 Apr 2026.

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