Axatilimab + ECP for Graft-versus-Host Disease

Overseen By Trent P Wang, DO

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Miami

Must be taking: Corticosteroids

Must not be taking: CSF-1R therapies

Disqualifiers: Pregnancy, Active malignancy, Pneumonitis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 7

Duration: 28 weeks

49 Participants Needed

Trial Summary

What is the purpose of 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).

Do I need to stop my current medications for the trial?

The trial requires that all GVHD systemic treatments be stopped except for corticosteroids and drugs being continued from GVHD prophylaxis at screening.

What data supports the effectiveness of the treatment Axatilimab + ECP for Graft-versus-Host Disease?

Extracorporeal photopheresis (ECP) has been shown to be effective in treating graft-versus-host disease (GvHD), especially in cases where other treatments like steroids have failed. It works by modulating the immune system, which can help manage the symptoms of GvHD. ^{1 2 3 4 5}

Is the combination of Axatilimab and Extracorporeal Photopheresis (ECP) safe for treating Graft-versus-Host Disease?

Extracorporeal Photopheresis (ECP) has been shown to be safe in patients with Graft-versus-Host Disease, with studies reporting no adverse effects related to ECP treatment. ^{4 6 7 8 9}

What makes the treatment Axatilimab + ECP unique for graft-versus-host disease?

Axatilimab + ECP is unique because it combines a novel drug, Axatilimab, with extracorporeal photochemotherapy (ECP), which is known for its dual ability to activate the immune system against cancer cells and reduce immune activity in autoimmune conditions. This combination may offer a new approach for managing graft-versus-host disease by leveraging these complementary effects. ^{2 4 10 11 12}

Research Team

Trent Wang, MD

Principal Investigator

University of Miami

Eligibility Criteria

This trial is for individuals with chronic Graft-versus-Host Disease (cGVHD), which can affect the skin and lungs after a stem cell or bone marrow transplant. Participants should have specific forms of cGVHD like skin disease or lung complications known as Bronchiolitis Obliterans Syndrome.

Inclusion Criteria

I am 12 years old or older.

I can care for myself but may not be able to do heavy physical work.

I am using birth control as required.

Exclusion Criteria

I do not have a lens in my eye due to surgery or a condition, or I have a disease that makes my skin or eyes very sensitive to light.

Pregnancy or breast-feeding

My cancer is currently getting worse.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Axatilimab in combination with extracorporeal photopheresis (ECP) therapy

28 weeks

7 cycles of 4 weeks each

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

Long-term follow-up

Participants are monitored for long-term safety and outcomes

15 months

Treatment Details

Interventions

• Axatilimab
• Extracorporeal Photopheresis

Trial Overview The study is testing the effectiveness of combining Axatilimab, a medication that targets immune cells, with Extracorporeal Photopheresis (ECP), a procedure where blood is treated with light to reduce immune activity, in treating cGVHD.

Participant Groups

Group I: Axatilimab in combination with ECP Group Experimental Treatment 2 Interventions

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.

Axatilimab is already approved in United States for the following indications:

Approved in United States as Axatilimab for:

• Chronic Graft-Versus-Host Disease

Who Is Running the Clinical Trial?

University of Miami

Lead Sponsor

Trials

976

Recruited

423,000+

Incyte Corporation

Industry Sponsor

Trials

408

Recruited

66,800+

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Long-term extracorporeal photoimmunotherapy for treatment of chronic cutaneous graft-versus-host disease: observations in four patients. [2017]

2. England pubmed.ncbi.nlm.nih.gov

Mechanism of action of extracorporeal photochemotherapy in chronic graft-versus-host disease. [2005]

3. United States pubmed.ncbi.nlm.nih.gov

Extracorporeal photopheresis versus anticytokine therapy as a second-line treatment for steroid-refractory acute GVHD: a multicenter comparative analysis. [2022]

4. New Zealand pubmed.ncbi.nlm.nih.gov

Systematic review and meta-analysis of prospective studies for ECP treatment in patients with steroid-refractory acute GVHD. [2020]

5. England pubmed.ncbi.nlm.nih.gov

Extracorporeal photoimmunotherapy for the treatment of steroid refractory progressive chronic graft-versus-host disease. [2006]

6. United States pubmed.ncbi.nlm.nih.gov

Extracorporeal photopheresis for GVHD prophylaxis after reduced intensity conditioning allogeneic hematopoietic stem cell transplantation: a prospective multicenter phase 2 study. [2019]

7. England pubmed.ncbi.nlm.nih.gov

Acute graft v host disease: developing an extracorporeal photopheresis outreach service. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

Extracorporeal photopheresis in solid organ transplant-associated acute graft-versus-host disease. [2016]

9. United States pubmed.ncbi.nlm.nih.gov

Phase II Trial of Costimulation Blockade With Abatacept for Prevention of Acute GVHD. [2022]

10. Italy pubmed.ncbi.nlm.nih.gov

Extracorporeal chemophototherapy for the treatment of graft-versus-host disease: hematologic consequences of short-term, intensive courses. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Immunomodulatory effects of extracorporeal photochemotherapy in patients with extensive chronic graft-versus-host disease. [2021]

12. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of one-day offline extracorporeal photopheresis schedule processing one total blood volume for treating patients with graft-versus-host disease. [2021]