Study of Efficacy and Safety of Ruxolitinib in Patients With Grade II to IV Steroid-refractory Acute Graft vs. Host Disease

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
high-dose systemic corticosteroids (methylprednisolone 2 mg/kg/day [or equivalent
prednisone dose 2.5 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 high-dose 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 high-dose
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 ≥2 mg/kg/day (or equivalent prednisone dose ≥2.5
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