This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.
Aged 2 to < 18 years at the time of randomization.
Active, moderate to severe cGVHD, requiring systemic immune suppression.
Participants with refractory or recurrent cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib.
Concomitant use of systemic corticosteroids is allowed. Participants on systemic corticosteroids must be on a stable dose of corticosteroids for at least 2 weeks prior to C1D1. Topical and inhaled corticosteroid agents are allowed.
Receipt of more than 1 prior allo-HCT. Prior autologous HCT is allowed, including autologous CAR T-cell therapy given before the allo-SCT.
Documented evidence of relapse of the primary hematologic disease or receipt of treatment for relapse after allo-SCT, including DLI for treatment of any malignancy relapse, including molecular relapse. Autologous and allogeneic donor-derived CAR T-cell therapy after allo-SCT are not allowed. Note: Participants who received DLI solely for the management of mixed chimerism or as part of the planned transplant procedure and not for treatment of malignancy relapse (including molecular relapse), are eligible.
Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
Severe renal impairment, that is, GFR < 30 mL/min/1.73 m2 as estimated using modified Schwartz formula, or end-stage renal disease on dialysis.
Parallel
INCA034176
DrugAxatilimab at the protocol-defined dose.
Best available Treatment (BAT)
DrugBest Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Objective Response (OR) at 6 months
Defined as complete response (CR) or partial response (PR) at 6 months (Cycle 7 Day 1, 28-day cycles) in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.
United States (US): Best overall Response (BOR)
Defined as a best response of CR or PR in the first 6 months (up to and including Cycle 7 Day 1) in the absence of new systemic therapy for cGVHD.
Pharmacokinetics Parameter (PK): Cmax of axatilimab
Defined as maximum observed plasma concentration of axatilimab.
Pharmacokinetics Parameter: Tmax of axatilimab
Defined as the time to reach the maximum plasma concentration of axatilimab.
Pharmacokinetics Parameter: Cmin of axatilimab
Defined as the minimum observed plasma concentration of axatilimab.
Pharmacokinetics Parameter: AUC(0-t) of axatilimab
Defined as the area under the concentration-time curve up to the last measurable concentration of axatilimab.
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