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Evaluating ruxolitinib for treating steroid-refractory acute graft versus host disease in Chinese patients

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

Phase 4 Interventional Novartis · NCT06462469

This study is testing if ruxolitinib can help Chinese adults and teens with severe graft versus host disease that hasn't responded to steroids.

Quick facts

PhasePhase 4
Study typeInterventional
Enrollment36 (estimated)
Ages12 Years to 100 Years
SexAll
SponsorNovartis Industry-sponsored
Drugs / interventionsruxolitinib, prednisone
Locations17 sites (Guangzhou, Guangdong and 16 other locations)
Trial IDNCT06462469 on ClinicalTrials.gov

What this trial studies

This study assesses the efficacy and safety of ruxolitinib in Chinese adults and adolescents aged 12 and older who have Grade II to IV steroid-refractory acute graft versus host disease (SR-aGvHD). Participants will undergo a screening period to determine eligibility, followed by treatment with ruxolitinib for 24 weeks. After treatment, safety follow-up visits will occur, with long-term follow-up extending to 12 months. The study aims to provide insights into the effectiveness of ruxolitinib for this challenging condition.

Who should consider this trial

Good fit: Ideal candidates are Chinese males and females aged 12 or older who have undergone allogeneic stem cell transplantation and have been diagnosed with Grade II to IV steroid-refractory acute graft versus host disease.

Not a fit: Patients who do not have steroid-refractory acute graft versus host disease or are younger than 12 years old may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a new therapeutic option for patients suffering from steroid-refractory acute graft versus host disease.

How similar studies have performed: Other studies have shown promising results with ruxolitinib for similar conditions, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion criteria

* 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:

  1. 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
  2. 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
  3. 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.

Key Exclusion criteria

* 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.

Other protocol-defined inclusion / exclusion criteria may apply

Where this trial is running

Guangzhou, Guangdong and 16 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Steroid-refractory Acute Graft Versus Host DiseaseSR-aGvHDaGvHDacute graft-versus-host diseaseruxolitinibChinese patientscorticosteroid-refractoryGrade II-IV
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.