Study of Efficacy and Safety of Ruxolitinib in Patients With Grade II to IV Steroid-refractory Acute Graft vs. Host Disease
A Single-arm, Multi-center Study of Ruxolitinib for the Treatment of Chinese Patients With Grade II-IV Corticosteroid-refractory Acute Graft Versus Host Disease
1 other identifier
interventional
36
1 country
17
Brief Summary
The purpose of this study is to assess the efficacy and safety of ruxolitinib therapy in Chinese adults and adolescents (≥ 12 years old) with Grade II-IV steroid-refractory acute graft versus host disease (SR-aGvHD).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_4
Started Jul 2024
Longer than P75 for phase_4
17 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 5, 2024
CompletedFirst Posted
Study publicly available on registry
June 17, 2024
CompletedStudy Start
First participant enrolled
July 4, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 4, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 17, 2028
November 25, 2025
November 1, 2025
2.5 years
June 5, 2024
November 23, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Overall Response Rate (ORR) at Day 28 per Investigators
The ORR at Day 28 defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without requirement for additional systemic therapies for an earlier progression, mixed response or nonresponse, according to standard criteria and assessed by investigators.
Day 28
Secondary Outcomes (10)
Durable Overall response rate (ORR) at Day 56
Day 56
Duration of Response (DOR)
From Week 1 to long term follow up Month 12
Best overall response (BOR)
From week 1 to Day 28
Overall survival (OS)
From the date of start of study treatment to date of death, up to approx. 12 months
Non-relapse mortality (NRM)
From date of start of study treatment to date of death, up to approx. 12 months
- +5 more secondary outcomes
Study Arms (1)
Ruxolitinib
EXPERIMENTALParticipants will receive ruxolitinib orally of 10 mg BID daily (given as two 5-mg tablets, approximately 12 hours apart).
Interventions
Ruxolitinib is taken orally daily at 10 mg BID, given as two 5-mg tablets.
Eligibility Criteria
You may qualify if:
- Male or female Chinese participants aged 12 or older at the time of informed consent. Written informed consent from participant, parent or legal guardian.
- Able to swallow tablets.
- Have undergone alloSCT from any donor source (matched unrelated donor, sibling, haplo-identical) using bone marrow, peripheral blood stem cells, or cord blood.
- Clinically diagnosed Grades II to IV acute GvHD as per standard criteria occurring after alloSCT requiring systemic immune suppressive therapy.
- Evident myeloid and platelet engraftment (confirmed within 48 hours prior to study treatment (ruxolitinib) start):
- Confirmed diagnosis of steroid refractory aGvHD defined as participants administered systemic corticosteroids (methylprednisolone at least 1 mg/kg/day \[or equivalent prednisone dose at least 1.25 mg/kg/day\]), given alone or combined with calcineurin inhibitors (CNI) and either:
- Progression based on organ assessment after at least 3 days compared to organ stage at the time of initiation of systemic corticosteroid +/- CNI for the treatment of Grade II to IV aGvHD. OR
- Failure to achieve at a minimum partial response based on organ assessment after 7 days compared to organ stage at the time of initiation of systemic corticosteroid +/-CNI for the treatment of Grade II to IV. OR
- Participants who fail corticosteroid taper defined as fulfilling either one of the following criteria:
- Requirement for an increase in the corticosteroid dose to methylprednisolone ≥ 1 mg/kg/day (or equivalent prednisone dose ≥ 1.25 mg/kg/day). OR
- Failure to taper the methylprednisolone dose to \< 0.5 mg/kg/day (or equivalent prednisone dose \<0.6 mg/kg/day) for a minimum of 7 days.
You may not qualify if:
- Has received more than one systemic treatment for steroid refractory aGvHD. Participants who received JAK inhibitor therapy for any indication after initiation of current alloSCT conditioning.
- Clinical presentation resembling de novo chronic GvHD or GvHD overlap syndrome with both acute and chronic GvHD features.
- Failed prior alloSCT within the past 6 months. Presence of relapsed primary malignancy after the alloSCT was performed.
- Presence of an active uncontrolled infection including significant bacterial, fungal, viral or parasitic infection requiring treatment.
- SR-aGvHD occurring after non-scheduled donor lymphocyte infusion (DLI) administered for pre-emptive treatment of malignancy recurrence. Note: Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.
- Presence of significant respiratory disease, severely impaired renal function, clinically significant or uncontrolled cardiac disease, unresolved cholestatic and liver disorders (not attributable to aGvHD). Disorders and/or current therapy with medications that interfere with coagulation or platelet function.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (17)
Novartis Investigative Site
Guangzhou, Guangdong, 510000, China
Novartis Investigative Site
Guangzhou, Guangdong, 510515, China
Novartis Investigative Site
Zhengzhou, Henan, 450003, China
Novartis Investigative Site
Wuhan, Hubei, 430030, China
Novartis Investigative Site
Changchun, Jilin, 130021, China
Novartis Investigative Site
Xian, Shanxi, 710061, China
Novartis Investigative Site
Chengdu, Sichuan, 610072, China
Novartis Investigative Site
Hangzhou, Zhejiang, 310003, China
Novartis Investigative Site
Beijing, 100028, China
Novartis Investigative Site
Beijing, 100034, China
Novartis Investigative Site
Beijing, 100039, China
Novartis Investigative Site
Beijing, 100070, China
Novartis Investigative Site
Dalian, 116000, China
Novartis Investigative Site
Fuzhou, 350001, China
Novartis Investigative Site
Shanghai, 200025, China
Novartis Investigative Site
Taian, 271099, China
Novartis Investigative Site
Tianjin, 300020, China
MeSH Terms
Interventions
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Central Study Contacts
Novartis Pharmaceuticals
CONTACT
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 5, 2024
First Posted
June 17, 2024
Study Start
July 4, 2024
Primary Completion (Estimated)
January 4, 2027
Study Completion (Estimated)
February 17, 2028
Last Updated
November 25, 2025
Record last verified: 2025-11
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.