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

March 29, 2026 updated by: Daihong Liu

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 )

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

54

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
      • Beijing, China, 100853
        • Recruiting
        • Department of Hematology, the Fifth Center of Chinese PLA General Hospital
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. diagnosed with hematological diseases.
  2. 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.
  3. New onset of severe acute GVHD within 100 days post-transplantation.

Exclusion Criteria:

  1. Recipients of second allogeneic stem cell transplant.
  2. Acute GVHD induced by donor lymphocyte infusion, interferon.
  3. Received first line aGVHD treatment before enrollment.
  4. Overlap GVHD syndrome.
  5. Pregnant or breast-feeding women.
  6. Absolute neutrophil count (ANC) <0.5×10e9/L or platelet count (PLT) < 20×10e9/L.
  7. Serum creatinine > 2.0 mg/dL or creatinine clearance < 40 mL/min measured or calculated by Cockroft-Gault equation.
  8. Uncontrolled infection.
  9. Human immunodeficiency virus infection.
  10. Active hepatitis b virus, hepatitis C virus infection and need antivirus treatment.
  11. Subjects with evidence of relapsed primary disease, or subjects who have been treated for relapse after the allo-HSCT was performed, or graft rejection.
  12. Allergic history to Janus kinase inhibitors.
  13. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ruxolitinib Arm
Ruxolitinib(5mg/d) combined with Methylprednisolone(1mg/kg)
Drug: Ruxolitinib and Methylprednisolone
Participants began oral administration of ruxolitinib at 5 mg QD; Methylprednisolone (1mg/kg)
Drug: Methylprednisolone
Methylprednisolone 2mg/kg/d , iv or iv gtt for at least 1 week, then taper according to the clinical response.
Active Comparator: Comparator arm
Methylprednisolone (2mg/kg)
Drug: Methylprednisolone
Methylprednisolone 2mg/kg/d , iv or iv gtt for at least 1 week, then taper according to the clinical response.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall response rate (ORR) at Day 28
Time Frame: Day 28 after treatment
Defined as the proportion of participants demonstrating a complete response (CR), and partial response (PR).
Day 28 after treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Six-month duration of response
Time Frame: Six-month after treatment
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.
Six-month after treatment
Nonrelapse mortality (NRM)
Time Frame: 1 year after treatment
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.
1 year after treatment
Ninety-day duration of response
Time Frame: Day 90 after treatment
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.
Day 90 after treatment
Cumulative incidence of relapse
Time Frame: 1 year after treatment
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.
1 year after treatment
Disease-free survival (DFS)
Time Frame: 1 year after treatment
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.
1 year after treatment
GVHD-free and relapse-free survival (GRFS)
Time Frame: 1 year after treatment
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.
1 year after treatment
recurrence of aGVHD
Time Frame: 1 year after treatment
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.
1 year after treatment
Failure-free survival
Time Frame: 1 year after treatment
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.
1 year after treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Daihong Liu

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 30, 2025

Primary Completion (Estimated)

December 30, 2027

Study Completion (Estimated)

December 30, 2027

Study Registration Dates

First Submitted

January 5, 2026

First Submitted That Met QC Criteria

March 29, 2026

First Posted (Actual)

April 3, 2026

Study Record Updates

Last Update Posted (Actual)

April 3, 2026

Last Update Submitted That Met QC Criteria

March 29, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • S2025-772-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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