A Phase II b Study of Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease
The purpose of this study is to see whether giving participants a combination treatment of Axatilimab and Extracorporeal Photopheresis (ECP) is effective against chronic Graft-versus-Host Disease (cGVHD).
• Recipient of allogeneic hematopoietic cell transplantation (HCT).
• Age greater or equal to 12.
• Chronic GVHD per 2014 National Institutes of Health Consensus Criteria (NCC) (Jagasia et al. 2015) or overlap syndrome requiring new therapy in patients with at least 2 prior lines of therapy, steroid refractoriness, or steroid dependence:
‣ Prior systemic lines of therapy may include corticosteroids, calcineurin inhibitor (CNI) or sirolimus, or other systemic immunosuppressive agent such as ruxolitinib, belumosudil, or ibrutinib. GVHD prophylaxis does not count as a prior line of therapy.
⁃ Steroid refractory is defined as any of the following criteria:
• i. Manifestations progress despite the use of ≥ 1 mg/kg/day prednisone for at least 1 week
∙ ii. Manifestations persist without improvement despite treatment with ≥ 0.5 mg/kg/day or 1 mg/kg every other day for at least four weeks.
∙ iii. Recurrence after a CR, or
∙ iv. Progression after a PR.
⁃ Steroid dependence is defined as inability to control cGVHD symptoms while tapering prednisone below 0.25 mg/kg/day on at least two occasions separated by at least 8 weeks. There must be evidence of clinically active cGVHD.
• For patients receiving approved or commonly used agents, all GVHD systemic treatments should be discontinued except for corticosteroids and drugs being continued from GVHD prophylaxis at screening.
• Eastern Cooperative Oncology Group (ECOG) performance status 0-3 as assessed at Screening.
• Platelet count \> 50,000 platelets/μL and absolute neutrophil count \> 1,000 cells/μL as measured at Screening.
• Aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤ 2.5 x upper limit of normal (ULN), unless attributed to presumed cGVHD as measured at Screening.
• Stable dose of corticosteroids for at least 14 days prior to treatment.
• Sexually mature individuals must use contraception as described in Section 4.12. For individuals less than 18 years of age, sexual maturity will be determined as per treating pediatrician.