About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Recipient of allogeneic hematopoietic cell transplantation (HCT).
Age greater or equal to 12.
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.
i. Manifestations progress despite the use of ≥ 1 mg/kg/day prednisone for at least 1 week
Disqualifiers
Pregnancy or breast-feeding.
Active relapse of underlying malignancy.
History or the presence of interstitial pneumonitis or drug-related pneumonitis.
Active gastrointestinal (GI) bleeding.
Trial design
Single group
Treatments tested in this trial
Axatilimab
Biological/VaccineAxatilimab will be administered intravenously (IV) at a dose of 0.3 mg/kg, beginning as a pre-phase dose two weeks prior to initiation of Extracorporeal Photopheresis (ECP) therapy. Thereafter, Axatilimab will be administered with a frequency of one treatment session bi-weekly during each treatment cycle.
Extracorporeal Photopheresis
Procedure/SurgeryMandatory ECP therapy will be administered at a frequency of two treatment sessions per week during Cycles 1 through 3, two treatment bi-weekly during Cycles 4 through 6, and two treatments during week 1 of Cycle 7. Optional ECP therapy will be administered at a frequency of two treatment sessions during weeks 2 and 4 of Cycles 4 through 6, when mandatory ECP is not administered. Optional ECP therapy will also be administered as two treatment sessions during week 3 of Cycle 7. After Cycle 7, participants may receive ECP therapy only at the Investigator's discretion for a maximum Treatment Period of 12 months.
Treatment groups
Trial outcomes
Primary outcomes
Best Overall Response Rate (ORR)
Best overall response rate (ORR) will be reported as the percentage of participants who achieve partial response (PR) or a complete response (CR) to study therapy, as defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in chronic graft-versus-host disease (cGVHD) while on study treatment.
Secondary outcomes
Proportion of participants experiencing treatment-related adverse events (AEs)
Proportion of participants experiencing treatment-related adverse events (AEs) will be reported. AEs, including serious adverse events (SAEs), will be assessed using the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0. Attribution of AEs and SAEs to study treatment will be reported as possibly, probably and definitely related, as determined by the treating physician.
Proportion of participants experiencing serious adverse events (SAEs)
Proportion of participants experiencing serious adverse events (SAEs) will be reported, regardless of attribution to study treatment, as determined by the treating physician. SAEs will be assessed using the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
Change in cumulative dose of corticosteroid usage
Change in cumulate dose corticosteroid usage among study participants will be reported, from baseline to 24 weeks and to 1-year post start of Axatilimab. Corticosteroid dosage, recorded as average dose in mg per day, will be recorded at each interval.
Duration of response (DOR)
Duration of response (DOR) is defined as the elapsed time in months from best response (partial response (PR) or complete response (CR)) to documented progression of chronic graft-versus-host disease (cGVHD), start of new therapy, or death for any reason.
Sponsors and contacts
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University of Miami
Lead sponsor
Incyte Corporation
Collaborator