Study of Efficacy and Safety of Ruxolitinib in Patients With Grade II to IV Steroid-refractory Acute Graft vs. Host Disease
- Conditions
- Steroid-refractory Acute Graft Versus Host Disease
- Registration Number
- NCT06462469
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 54
Key Inclusion criteria<br><br> - Male or female Chinese participants aged 12 or older at the time of informed<br> consent. Written informed consent from participant, parent or legal guardian.<br><br> - Able to swallow tablets.<br><br> - Have undergone alloSCT from any donor source (matched unrelated donor, sibling,<br> haplo-identical) using bone marrow, peripheral blood stem cells, or cord blood.<br><br> - Clinically diagnosed Grades II to IV acute GvHD as per standard criteria occurring<br> after alloSCT requiring systemic immune suppressive therapy.<br><br> - Evident myeloid and platelet engraftment (confirmed within 48 hours prior to study<br> treatment (ruxolitinib) start):<br><br> - Confirmed diagnosis of steroid refractory aGvHD defined as participants administered<br> high-dose systemic corticosteroids (methylprednisolone 2 mg/kg/day [or equivalent<br> prednisone dose 2.5 mg/kg/day]), given alone or combined with calcineurin inhibitors<br> (CNI) and either:<br><br> 1. Progression based on organ assessment after at least 3 days compared to organ<br> stage at the time of initiation of high-dose systemic corticosteroid +/- CNI<br> for the treatment of Grade II to IV aGvHD. OR<br><br> 2. Failure to achieve at a minimum partial response based on organ assessment<br> after 7 days compared to organ stage at the time of initiation of high-dose<br> systemic corticosteroid +/-CNI for the treatment of Grade II to IV. OR<br><br> 3. Participants who fail corticosteroid taper defined as fulfilling either one of<br> the following criteria:<br><br> - Requirement for an increase in the corticosteroid dose to<br> methylprednisolone =2 mg/kg/day (or equivalent prednisone dose =2.5<br> mg/kg/day). OR<br><br> - Failure to taper the methylprednisolone dose to < 0.5 mg/kg/day (or<br> equivalent prednisone dose <0.6 mg/kg/day) for a minimum of 7 days.<br><br>Key Exclusion criteria<br><br> - Has received more than one systemic treatment for steroid refractory aGvHD.<br> Participants who received JAK inhibitor therapy for any indication after initiation<br> of current alloSCT conditioning.<br><br> - Clinical presentation resembling de novo chronic GvHD or GvHD overlap syndrome with<br> both acute and chronic GvHD features.<br><br> - Failed prior alloSCT within the past 6 months. Presence of relapsed primary<br> malignancy after the alloSCT was performed.<br><br> - Presence of an active uncontrolled infection including significant bacterial,<br> fungal, viral or parasitic infection requiring treatment.<br><br> - SR-aGvHD occurring after non-scheduled donor lymphocyte infusion (DLI) administered<br> for pre-emptive treatment of malignancy recurrence. Note: Participants who have<br> received a scheduled DLI as part of their transplant procedure and not for<br> management of malignancy relapse are eligible.<br><br> - Presence of significant respiratory disease, severely impaired renal function,<br> clinically significant or uncontrolled cardiac disease, unresolved cholestatic and<br> liver disorders (not attributable to aGvHD). Disorders and/or current therapy with<br> medications that interfere with coagulation or platelet function.<br><br>Other protocol-defined inclusion / exclusion criteria may apply
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Overall Response Rate (ORR) at Day 28 per Investigators
- Secondary Outcome Measures
Name Time Method Durable Overall response rate (ORR) at Day 56;Duration of Response (DOR);Best overall response (BOR);Overall survival (OS);Non-relapse mortality (NRM);Event-free survival (EFS);Failure-free survival (FFS);Malignancy Relapse/Progression (MR);Reduction of daily corticosteroids dose;Cumulative incidence of chronic GvHD