steroid refractory chronic graft versus host disease(SR-cGvHD)
Conditions
Interventions
- Ruxolitinib will be administered to patients randomized to the study drug treatment arm at a starting dose of 10 mg orally BID, without regard to food.
- The BAT in this study will be selected by th
Sponsors
Novartis Pharma. K.K.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female patients =>12 years old at the time of signing the ICF - 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) > 1000/mm3 and platelet count > 25,000/ mm3 - Patients with clinically diagnosed moderate to severe cGvHD according to NIH Consensus Criteria (Jagasia 2015) prior to randomization: - 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 - Patients currently receiving systemic or topical corticosteroids for the treatment of cGvHD for a duration of 0.5 mg/kg/day or 1 mg/kg/every other day for at least 4 weeks (or equivalent), OR - Increase to prednisolone dose to >0.25 mg/kg/day after two unsuccessful attempt to taper the dose (or equivalent) - Patient must accept to be treated with only one of the following BAT options on Cycle 1 Day 1. (Additions and changes are allowed during the course of the study, but only with BAT from the following BAT options): extracorporeal photopheresis (ECP), low-dose methotrexate (MTX), mycophenolate mofetil (MMF), mTOR inhibitors (everolimus or sirolimus), infliximab, rituximab, pentostatin, imatinib, ibrutinib
Exclusion criteria
Exclusion criteria: - Patients who have received two or more systemic treatments for cGvHD in addition to corticosteroids +/- CNI for cGvHD - Patients that transition from active aGvHD to cGvHD without tapering off corticosteroids +/- CNI and any systemic treatment - Patients who were treated with prior JAK inhibitors for aGvHD; except when the patient 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 - Patients 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. Patients who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible - Any corticosteroid therapy for indications other than cGvHD at doses >1 mg/kg/day methylprednisolone or equivalent within 7 days of Cycle 1 Day 1
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy To compare the efficacy of ruxolitinib vs. Investigator choice Best Available Therapy (BAT) in patients with moderate or severe SR-cGvHD assessed by Overall Response Rate (ORR) at the Cycle 7 Day 1 visit | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy To compare the rate of failure free survival (FFS) | — |
Countries
Asia except Japan, Europe, Japan, North America, Oceania
Contacts
Public ContactTakamitsu Hirano
Novartis Pharma. K.K.
Outcome results
None listed