RecruitingPHASE3INTERVENTIONAL

A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease

This research study is investigating a new treatment, axatilimab, for people who have chronic graft-versus-host disease (cGVHD) that has recently developed or become worse. cGVHD is a complication that can happen after a stem cell transplant, where the donated immune cells attack the patient's own body. The study will compare axatilimab, given with corticosteroids (steroids), against a dummy medicine (placebo) also given with corticosteroids. The main goal is to find out if axatilimab is an effective and safe first-time treatment for moderate or severe cGVHD. This is a Phase 3 study, meaning it's a large trial testing the new treatment against existing options or placebo to confirm its benefits and side effects.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Incyte Corporation

Enrolment target

240

Start

21 Jan 2025

Estimated completion

31 Mar 2030

Chronic Graft-versus-host-disease

What is this study about?

This study is for people who have a condition called chronic graft-versus-host disease, often shortened to cGVHD. This can sometimes happen after a special type of transplant called an allogeneic haematopoietic cell transplant (allo-HCT). During this transplant, healthy cells from a donor are given to a patient to help treat certain diseases. In cGVHD, the donor cells start to recognise the patient's body as 'foreign' and begin to attack it, which can cause various health problems.

The trial is looking at a new medicine called axatilimab. It will be tested alongside corticosteroids, which are a common type of medicine used to treat cGVHD. Some participants will receive axatilimab plus corticosteroids, while others will receive a placebo (a dummy medicine with no active ingredients) plus corticosteroids. This comparison helps researchers understand if axatilimab offers additional benefits over corticosteroids alone for people with moderate or severe cGVHD that has recently started or requires treatment.

The aim is to see if axatilimab can be an effective and safe initial treatment. 'Initial treatment' means it would be one of the first medicines used when someone develops cGVHD or when their cGVHD gets worse. By carefully comparing the two groups, doctors hope to learn if this new medicine could become a valuable option to help manage cGVHD and improve the lives of those affected.

Key takeaways

The study explores a new medicine (axatilimab) for chronic GVHD.
It's for people aged 12+ with new or worsening moderate/severe cGVHD.
Participants will receive either axatilimab or a placebo, plus standard steroids.
The aim is to find effective first-time treatments for cGVHD.
Close medical monitoring is provided throughout the study.

Who may be eligible?

This study is looking for people aged 12 and older who have been newly diagnosed with moderate or severe chronic graft-versus-host disease (cGVHD) that needs treatment. You must have had an allogeneic stem cell transplant in the past, no matter where the cells came from or how they were matched.

There are also some reasons why you might not be able to join. For instance, if you've had more than one stem cell transplant, or if you already have a mix of acute and chronic GVHD. You also can't have received much steroid treatment for your cGVHD before or have tried other specific cGVHD treatments. The study also cannot include those with ongoing severe infections or if their original blood disease has come back.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 12 years old or older?
Have you recently been diagnosed with moderate or severe cGVHD requiring treatment?
Have you had a stem cell transplant in the past?
Have you NOT received more than a short course of steroid treatment for cGVHD recently?
Do you NOT have an active, uncontrolled infection or your original blood disease back?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive either the new medicine (axatilimab) or a placebo, both given with corticosteroids, as your main treatment for cGVHD. You'll have regular hospital visits for check-ups, blood tests, and to receive your study medication. These visits will help doctors monitor your health, how your cGVHD is responding, and if you have any side effects. The total duration of the study and how often you'll need to visit will be explained in detail by the study team, but typically, these types of trials involve regular follow-ups for a number of months or even longer until the treatment period ends and your health is stable.

Potential risks and benefits

Taking part in a clinical trial might offer potential benefits, such as gaining access to a new treatment before it's widely available and receiving close medical attention for your condition. However, there are also potential risks, including side effects from the study medication, which may or may not be known yet. It's important to remember that the new treatment might not work for everyone, or it might not be better than existing treatments. You will be given full details of known and potential risks and benefits. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (122)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Alabama Birmingham
Verified postcode
Birmingham, United States· Recruiting
University of California San Diego Medical Center, Moores Cancer Center
Verified postcode
La Jolla, United States· Recruiting
University of Southern California
Verified postcode
Los Angeles, United States· Recruiting
Colorado Blood Cancer Institute
Verified postcode
Denver, United States· Recruiting
Childrens National Hospital
Verified postcode
Washington D.C., United States· Recruiting
Miami Cancer Institute
Verified postcode
Miami, United States· Recruiting
Orlando Health Cancer Institute Downtown Orlando
Verified postcode
Orlando, United States· Recruiting
Memorial Cancer Institute
Verified postcode
Pembroke Pines, United States· Recruiting
Emory University-Winship Cancer Institute
Verified postcode
Atlanta, United States· Recruiting
Northwestern Memorial Hospital
Verified postcode
Chicago, United States· Recruiting
University of Illinois
Verified postcode
Chicago, United States· Recruiting
The University of Kansas Cancer Center
Verified postcode
Kansas City, United States· Recruiting

Common questions

What is chronic graft-versus-host disease (cGVHD)?

cGVHD is a complication after a stem cell transplant where the new immune cells mistakenly attack the patient's own body tissues.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the active drug but contains no active ingredients. It's used for comparison in studies.

How long will I be in the study?

The exact length of time you'll be involved will be explained by the study team, but it typically involves several months of treatment and follow-up.

Will I know if I'm getting the new medicine or the placebo?

No, this type of study is 'blinded,' meaning neither you nor your doctors will know which treatment you are receiving until after the study is complete, unless there's a medical emergency.

Can I leave the study if I change my mind?

Yes, you are free to leave the study at any time without having to give a reason, and it will not affect your ongoing medical care.

How to find out more

Incyte Corporation Call Center (US)

medinfo@incyte.com 1.855.463.3463 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.