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.
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
- Conditions the study is for
- GVHD, Acute, Stem Cell Transplant Complications
- What is being tested?
- Ruxolitinib and Methylprednisolone and Methylprednisolone
- Type of treatment
- Pill or oral medicine, Injection or infusion
- 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
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.
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- 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.
China1 site, 1 recruiting
- 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
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.
- Conditions
- GVHD, Acute, Stem Cell Transplant Complications
- 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)
Methylprednisolone 2mg/kg/d , iv or iv gtt for at least 1 week, then taper according to the clinical response.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib Arm | Experimental | Ruxolitinib(5mg/d) combined with Methylprednisolone(1mg/kg) Receives: Drug: Ruxolitinib and Methylprednisolone; Drug: Methylprednisolone |
| Comparator arm | Active comparator | Methylprednisolone (2mg/kg) Receives: Drug: Methylprednisolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
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
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
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
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
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
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
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
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.
- First posted3 Apr 2026
- Study start30 Jun 2025
- Primary completion30 Dec 2027 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for GVHD, AcuteStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Daihong Liu
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Daihong Liu, Chinese PLA General Hospital
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 29382747Zeiser 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 32320566Michonneau 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 38804281Mehta 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 39936555Dou 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