Study of Efficacy and Safety of Ruxolitinib in Chinese Participants With Corticosteroid-refractory Chronic Graft vs. Host Disease

This is a single arm, multi-center, open label study which will enroll approximately 50 participants and investigate the efficacy and safety of ruxolitinib administered in adult and adolescent (≥12 years old) Chinese participants with SR-cGvHD.

The total duration on study for an individual participant will be up to 164 weeks (approximately 3 years).

The study consists of following periods, with each cycle comprised of 4 weeks (28 days):

• Screening Period (Day -28 to Day -1)
• Treatment period (Day 1 to Cycle 39/EOT)
• Safety follow-up (Last dose +30 days)
• Long-term survival follow-up period (EOT to 156 weeks on study).

Inclusion criteria

• Signed informed consent must be obtained prior to participation in the study.
• Male or female Chinese participants aged 12 or older at the time of informed consent
• 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. Recipients of non-myeloablative, myeloablative, and reduced intensity conditioning are eligible.
• Evident myeloid and platelet engraftment:

• Absolute neutrophil count (ANC) >1,000/mm3 AND
• Platelet count ≥25,000/mm3

Note: Use of growth factor supplementation and transfusion support is allowed during the trial, however, transfusion to reach a minimum platelet count for inclusion is not allowed during screening and at baseline.

• Participants with clinically diagnosed cGvHD staging of moderate to severe according to NIH Consensus Criteria (Jagasia et al 2015) prior to Cycle 1 Day 1.

• Moderate cGvHD: at least one organ (not lung) with a score of 2, 3 or more organs involved with a score of 1 in each organ, or lung score of 1.
• Severe cGvHD: at least 1 organ with a score of 3, or lung score of 2 or 3.
• Participants currently receiving systemic corticosteroids for the treatment of cGvHD for a duration of < 12 months prior to Cycle 1 Day 1, and have a confirmed diagnosis of corticosteroid refractory cGvHD defined per 2014 NIH consensus criteria (Martin et al 2015) irrespective of the concomitant use of a calcineurin inhibitor, as follows:

• A lack of response or disease progression after administration of minimum prednisone 1 mg/kg/day for at least 1 week (or equivalent) OR
• Disease persistence without improvement despite continued treatment with prednisone at >0.5 mg/kg/day or 1 mg/kg/every other day for at least 4 weeks (or equivalent) OR
• Increase to prednisone dose to >0.25 mg/kg/day after two unsuccessful attempts to taper the dose (or equivalent)
• Participants has Eastern Cooperative Oncology Group (ECOG) performance status of 0-2

Exclusion criteria

For a full list of exclusion criteria, refer to Section 5.2. Key exclusion criteria include

• Participants who have received two or more systemic treatments for cGvHD in addition to corticosteroids ± CNI for cGvHD.
• Participants who have received ROCK2 inhibitors for cGvHD.
• Participants that transition from active aGvHD to cGvHD without tapering off corticosteroids ± CNI and any systemic treatment

Note: Participants receiving up to 30 mg by mouth once a day of hydrocortisone (i.e., physiologic replacement dose) of corticosteroids are allowed.

• Participants who were treated with prior JAK inhibitors for aGvHD; except when the participant achieved complete or partial response and has been off JAK inhibitor treatment for at least 8 weeks prior to Cycle 1 Day 1.
• Failed prior alloSCT within the past 6 months from Cycle 1 Day 1.
• Participants with relapsed primary malignancy, or who have been treated for relapse after the alloSCT was performed.
• SR-cGvHD occurring after a non-scheduled donor lymphocyte infusion (DLI) administered for pre-emptive treatment of malignancy recurrence. Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.

Other protocol-defined inclusion/exclusion may apply.