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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment combination for individuals with chronic Graft-versus-Host Disease (cGVHD), a condition that can occur after a stem cell transplant when the donor's cells attack the recipient's body. Researchers aim to determine if Axatilimab (an experimental treatment) combined with Extracorporeal Photopheresis (a process where blood is treated with light) effectively treats this condition. Individuals who have undergone at least two previous treatments for cGVHD without success, or who cannot reduce their steroid use without symptoms returning, might consider joining. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that Extracorporeal Photopheresis (ECP) is a safe treatment for people with chronic Graft-versus-Host Disease (cGVHD). Studies have found ECP to be both safe and effective, with only minor side effects reported.

Regarding Axatilimab, there is some encouraging information. A previous study showed a good response rate in patients with cGVHD who had not succeeded with other treatments. While this study focused on efficacy, earlier research suggests that Axatilimab is generally safe and well-tolerated.

Since this trial is in an early stage, earlier studies likely indicate a reasonable safety profile, but ongoing research is important to confirm these results. Participants should be informed of any potential risks and are encouraged to discuss any concerns with the trial organizers. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for chronic Graft-versus-Host Disease?

Most treatments for graft-versus-host disease (GVHD) focus on suppressing the immune system to reduce the attack on the host's tissues. However, Axatilimab is unique because it targets the CSF-1 receptor, which plays a key role in the survival and function of monocytes and macrophages, the cells involved in GVHD. Researchers are excited about Axatilimab because it offers a new mechanism of action, potentially reducing GVHD symptoms without broadly suppressing the entire immune system. This specific targeting could lead to fewer side effects and improved outcomes for patients compared to standard immunosuppressive therapies.

What evidence suggests that this combination treatment is effective for chronic Graft-versus-Host Disease?

Research has shown that Axatilimab may help treat chronic Graft-versus-Host Disease (cGVHD). One study found that 74% of patients who had tried several other treatments responded well to Axatilimab. Extracorporeal Photopheresis (ECP) is already recognized as a safe and effective treatment for GvHD. This trial will combine Axatilimab with ECP to evaluate whether this combination can enhance treatment outcomes. These findings suggest that this combination could benefit people dealing with cGVHD. ^{1 2 3 4 6}

Who Is on the Research Team?

Trent Wang, MD

Principal Investigator

University of Miami

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06663722

Study Details | NCT06663722 | Axatilimab in Combination ... The purpose of this study is to see whether giving participants a combination treatment of Axatilimab and Extracorporeal Photopheresis (ECP) is effective ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06663722?term=AREA%5BConditionSearch%5D(%22Chronic%20Graft%20Versus%20Host%20Disease%22)&rank=7

Axatilimab in Combination With Extracorporeal ... The purpose of this study is to see whether giving participants a combination treatment of Axatilimab and Extracorporeal Photopheresis (ECP) is effective ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11510389/

Safety and Efficacy of Extracorporeal Photopheresis for ... Conclusions: Our results confirm the safety and efficacy of ECP in patients with GvHD and provide sufficient data for further investigation and ...

4. pharmacytimes.com pharmacytimes.com/view/axatilimab-a-new-frontier-for-steroid-refractory-chronic-gvhd

Axatilimab: A New Frontier for Steroid-Refractory Chronic ... The AGAVE-201 trial showed a 74% overall response rate for axatilimab in heavily pretreated cGVHD patients.

5. sciencedirect.com sciencedirect.com/science/article/pii/S266663672501156X

Changes in Chronic Graft-versus-Host Disease Treatment ... Chronic graft-versus-host disease (cGVHD) represents a major complication after allogeneic stem cell transplantation (alloHSCT). In 2009 and 2018 a survey ...

6. ascopubs.org ascopubs.org/doi/pdfdirect/10.1200/jco.22.00958

Axatilimab for Chronic Graft-Versus-Host Disease After ... Here, we present the primary analysis of a phI/II study of axatilimab and describe its safety and efficacy in a heavily pretreated patient ...