Actively Recruiting
A Phase IIb Study of Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease
Led by University of Miami · Updated on 2026-05-15
49
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
U
University of Miami
Lead Sponsor
I
Incyte Corporation
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating whether a combination treatment of Axatilimab and Extracorporeal Photopheresis (ECP) is effective against chronic Graft-versus-Host Disease (cGVHD). This Phase IIb study focuses on patients with cGVHD who have had at least two prior therapies or who are steroid refractory or dependent, aiming to assess the best overall response rate within 24 weeks. Participants will receive Axatilimab intravenously at a dose of 0.3 mg/kg, starting with a pre-phase dose two weeks before beginning ECP therapy. Axatilimab treatments occur bi-weekly during each cycle. ECP therapy is mandatory twice weekly during the first three cycles, then bi-weekly in cycles 4 to 6, with specific sessions in cycle 7 and optional sessions as needed. Treatment lasts up to seven four-week cycles, with possible extended ECP therapy for up to 12 months at the investigator's discretion. Over about 15 months, participants will be assessed for treatment response, safety, changes in corticosteroid use, quality of life, and relapse-free survival. Researchers will monitor adverse events, serious adverse events, symptom scores, and the development of sclerotic skin disease. Regular evaluations include clinical assessments and symptom scales to track the treatment's impact and safety throughout the study period.
CONDITIONS
Brief Title
Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Recipient of allogeneic hematopoietic cell transplantation (HCT)
- Age 12 years or older
- Diagnosis of chronic GVHD per 2014 National Institutes of Health Consensus Criteria or overlap syndrome requiring new therapy with at least 2 prior lines of therapy, steroid refractoriness, or steroid dependence
- Prior systemic treatments may include corticosteroids, calcineurin inhibitors, sirolimus, ruxolitinib, belumosudil, or ibrutinib; GVHD prophylaxis does not count
- Steroid refractory defined as progression or persistence despite prednisone use as specified, or recurrence/progression after response
- Steroid dependence defined as inability to taper prednisone below 0.25 mg/kg/day on at least two occasions separated by at least 8 weeks with active cGVHD
- Discontinuation of all GVHD systemic treatments except corticosteroids and certain prophylaxis drugs at screening
- ECOG performance status 0-3 at screening
- Platelet count > 50,000/µL and absolute neutrophil count > 1,000 cells/µL at screening
- AST and ALT ≤ 2.5 times upper limit of normal unless due to cGVHD
- Stable corticosteroid dose for at least 14 days prior to treatment
- Sexually mature individuals must use contraception as described
You will not qualify if you...
- Pregnancy or breast-feeding
- Active relapse of underlying malignancy
- History or presence of interstitial pneumonitis or drug-related pneumonitis
- Active gastrointestinal bleeding
- Inability to tolerate volume shifts from ECP due to inadequate organ function or cardiac ejection fraction < 40%
- History of myositis
- History of splenectomy
- History of pancreatitis
- History of other malignancy within 3 years unless treated with curative intent and approved
- Significant uncontrolled or active comorbid conditions or inability to adhere to study requirements
- AIDS or active hepatitis B or C infection
- Prior colony-stimulating factor-1 receptor targeted therapies
- Prior ECP treatment failure or intolerance
- Intolerance to methoxsalen, heparin, or citrate products
- Patients with aphakia or photosensitive diseases like albinism, lupus, or porphyria
- Lack of stable intravenous access
- Insurance denial of coverage for ECP procedure
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
screening and enrollment visit
Duration - Up to 7 four-week cycles (approximately 7 months)
Participants receive Axatilimab intravenously combined with Extracorporeal Photopheresis (ECP) therapy through up to seven 4-week cycles. Axatilimab starts with a pre-phase dose two weeks before ECP and is given bi-weekly during treatment cycles. ECP therapy frequency varies during treatment cycles with mandatory and optional sessions.
Multiple treatment sessions weekly and bi-weekly over 7 cycles
Duration - Up to 12 months after initial treatment
Participants may continue to receive ECP therapy at the Investigator's discretion for a total treatment period of up to 12 months. Safety and response are monitored during this period.
Visits as determined by investigator for continued ECP therapy and monitoring
Trial Site Locations
Total: 1 location
1
University of Miami
Miami, Florida, United States, 33136
Actively Recruiting
Research Team
T
Trent P Wang, DO
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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