Novel First-line Therapies for Grade II Acute GVHD(Graft-versus-host Disease )

NCT07340723 | Recruiting Phase 2 DMC

• 1 other identifier: S2025-767-01
• Study Type: interventional
• Target: 168
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

GVHD,Acute; Stem Cell Transplant Complications

Keywords

acute graft-versus-host disease; Ruxolitinib; first line therapy

Outcome Measures

Primary Outcomes (1)

• Overall response rate (ORR) at Day 28

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

  Day 28 after treatment

Secondary Outcomes (9)

• Six-month duration of response

  Six-month after treatment

• Duration of response

  Day 90 after treatment

• Nonrelapse mortality (NRM)

  1 year after treatment

• Cumulative incidence of relapse (CIR)

  1 year after treatment

• Disease-free survival (DFS)

  1 year after treatment

Study Arms (2)

Ruxolitinib Arm

EXPERIMENTAL

Ruxolitinib combined with Corticosteroids

Drug: Ruxolitinib; Drug: Corticosteroids

Comparator arm

ACTIVE COMPARATOR

Drug: Corticosteroids

Interventions

Ruxolitinib DRUG

Participants began oral administration of ruxolitinib at 5 mg QD;Methylprednisolone: 0.5mg/kg/d , iv or iv gtt for at least 5 days, then taper according to the clinical response.

Ruxolitinib Arm

Corticosteroids DRUG

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

Comparator arm; Ruxolitinib Arm

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 grade II acute GVHD or intermediate or high risk aGVHD (based on modified GVHD Glucksberg criteria) within 100 days post-transplantation.

You may not qualify if:

• 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.
• Pregnant or breast-feeding women.
• 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.

Sponsors & Collaborators

• Daihong Liu: lead

Study Sites (1)

Department of Hematology, Senior Department of Hematology, The Fifth Medical Center of PLA General Hospital

Beijing, Beijing Municipality, 100853, China

RECRUITING

Related Publications (5)

• von Bubnoff N, Ihorst G, Grishina O, Rothling N, Bertz H, Duyster J, Finke J, Zeiser R. Ruxolitinib in GvHD (RIG) study: a multicenter, randomized phase 2 trial to determine the response rate of Ruxolitinib and best available treatment (BAT) versus BAT in steroid-refractory acute graft-versus-host disease (aGvHD) (NCT02396628). BMC Cancer. 2018 Nov 19;18(1):1132. doi: 10.1186/s12885-018-5045-7.

  PMID: 30453910 BACKGROUND

• 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.

  PMID: 32320566 BACKGROUND

• Kujawska J, Zeiser R, Gil L. Recent advances in acute gastrointestinal graft versus host disease (aGvHD): aspects of steroid-resistant disease. Ann Hematol. 2025 Feb;104(2):855-865. doi: 10.1007/s00277-024-05952-0. Epub 2024 Aug 29.

  PMID: 39207560 BACKGROUND

• Holtan SG, Yu J, Choe HK, Paranagama D, Tang J, Naim A, Galvin J, Joachim Deeg H. Disease progression, treatments, hospitalization, and clinical outcomes in acute GVHD: a multicenter chart review. Bone Marrow Transplant. 2022 Oct;57(10):1581-1585. doi: 10.1038/s41409-022-01764-w. Epub 2022 Jul 30.

  PMID: 35908108 BACKGROUND

• 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.

  PMID: 39438467 BACKGROUND

MeSH Terms

Interventions

ruxolitinib; Adrenal Cortex Hormones

Intervention Hierarchy (Ancestors)

Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists

Central Study Contacts

Liping Dou, M.D., Ph.D.

CONTACT

010-66937079; lipingruirui@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Department of Hematology, Senior Department of Hematology, The Fifth Medical Center of PLA General Hospital

Model Details: Drug:Ruxolitinib, Corticosteroids

Study Record Dates

• First Submitted: January 5, 2026
• First Posted: January 14, 2026
• Study Start: June 1, 2025
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027
• Last Updated: January 14, 2026

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)