Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease

NCT06663722 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 20240116
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 1

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).

Conditions

Chronic Graft Versus Host Disease; cGVHD

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 24 weeks

Secondary Outcomes (8)

• Proportion of participants experiencing treatment-related adverse events (AEs)

  Up to 15 months

• 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

Study Arms (1)

Axatilimab in combination with ECP Group

EXPERIMENTAL

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.

Biological: Axatilimab; Procedure: Extracorporeal Photopheresis

Interventions

Axatilimab BIOLOGICAL

Axatilimab 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.

Axatilimab in combination with ECP Group

Extracorporeal Photopheresis PROCEDURE

Mandatory 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.

Also known as: ECP

Axatilimab in combination with ECP Group

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Acquired Immune Deficiency Syndrome (AIDS) or active hepatitis B (Hep B) or active hepatitis C (Hep C) infection.
• Prior colony-stimulating factor-1 (CSF-1R) targeted therapies.
• Prior history of ECP treatment failure or intolerance.
• Intolerance to methoxsalen, heparin, or citrate products.
• Patients with aphakia due to risk of increased retinal damage or photosensitive disease (albinism, systemic lupus erythematosus, porphyria).

Sponsors & Collaborators

• University of Miami: lead
• Incyte Corporation: collaborator

Study Sites (1)

University of Miami

Miami, Florida, 33136, United States

RECRUITING

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

axatilimab; Photopheresis

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Intervention Hierarchy (Ancestors)

PUVA Therapy; Ultraviolet Therapy; Phototherapy; Therapeutics; Extracorporeal Circulation; Surgical Procedures, Operative

Study Officials

• Trent P Wang, DO

  University of Miami

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Trent P Wang, DO

CONTACT

+1 (305) 2436444; trentwang@med.miami.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 26, 2024
• First Posted: October 29, 2024
• Study Start: May 5, 2025
• Primary Completion (Estimated): May 5, 2030
• Study Completion (Estimated): May 5, 2030
• Last Updated: May 20, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)