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

Launched by NOVARTIS PHARMACEUTICALS · Feb 10, 2025

Keywords

ClinConnect Summary

This clinical trial is investigating the effectiveness and safety of a medication called ruxolitinib for Chinese adults and children aged 12 and older who have a specific condition known as corticosteroid-refractory chronic graft vs. host disease (SR-cGvHD). This condition can occur after a stem cell transplant, where the transplanted cells attack the recipient's body, and it can be difficult to treat with standard steroid medications. The study aims to find out if ruxolitinib can help manage this disease in participants who haven't responded well to corticosteroids.

To be eligible for the trial, participants must be at least 12 years old, have a confirmed diagnosis of moderate to severe cGvHD, and have been treated with corticosteroids without improvement in their condition. Participants need to be able to swallow tablets and have a certain level of blood cell recovery from their transplant. Those interested should be aware that the trial is not yet recruiting, but it will involve regular monitoring and follow-up during the study. This research hopes to provide new treatment options for individuals struggling with this challenging condition.

Gender

ALL

Eligibility criteria

• 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.