A Phase II b Study of Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease

University of Miami1 site in 1 country49 target enrollmentMay 5, 2025

ConditionsChronic Graft Versus Host Disease cGVHD

InterventionsAxatilimab Extracorporeal Photopheresis

DrugsAxatilimab

Overview

Phase: Phase 2
Intervention: Axatilimab
Conditions: Chronic Graft Versus Host Disease
Sponsor: University of Miami
Enrollment: 49
Locations: 1
Primary Endpoint: Best Overall Response Rate (ORR)
Status: Recruiting
Last Updated: 11 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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).

Registry

clinicaltrials.gov

Start Date

May 5, 2025

End Date

May 5, 2030

Last Updated

11 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

University of Miami

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Recipient of allogeneic hematopoietic cell transplantation (HCT).
•Age greater or equal to
•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.

Exclusion Criteria

•Pregnancy or breast-feeding.
•Active relapse of underlying malignancy.
•History or the presence of interstitial pneumonitis or drug-related pneumonitis.
•Active gastrointestinal (GI) bleeding.
•Inability to tolerate volume shifts associated with ECP (e.g., inadequate renal, hepatic, pulmonary and cardiac function (ejection fraction (EF) \< 40%) per Investigator discretion.
•History of myositis.
•History of splenectomy.
•History of pancreatitis.
•History of other malignancy (within 3 years of Screening) unless treated with curative intent and approved by Principal Investigator (PI).
•Significant, uncontrolled, or active comorbid conditions or are unable to adhere to the study requirements.

Arms & Interventions

Axatilimab in combination with ECP Group

Participants in this group will receive Axatilimab in combination with extracorporeal photopheresis (ECP) therapy for up to seven (7) four-week cycles. Total participation duration is about 15 months.

Intervention: Axatilimab

Axatilimab in combination with ECP Group

Intervention: Extracorporeal Photopheresis

Outcomes

Primary Outcomes

Best Overall Response Rate (ORR)

Time Frame: Up to 24 weeks

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 serious adverse events (SAEs)(Up to 15 months)
Change in cumulative dose of corticosteroid usage(Baseline, 24 weeks, 1 year)
Duration of response (DOR)(Up to 15 months)
Relapse-free survival (RFS)(Up to 15 months)
Proportion of participants experiencing treatment-related adverse events (AEs)(Up to 15 months)
Change in Quality of life (QoL) as measured by the modified Lee Symptom Scale (mLSS) score(Baseline, 24 weeks, 1 year)
Proportion of participants who develop subsequent sclerotic skin disease(Up to 15 months)
Rate of Complete Response (CR) at Best Response(Up to 24 weeks)