Active not recruitingPHASE2INTERVENTIONAL

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)

This study is looking at a new medicine, axatilimab, for people with chronic graft-versus-host disease (cGVHD). This condition can happen after a stem cell transplant when the donor cells attack the patient's body. The study focuses on people whose cGVHD has come back or hasn't improved after at least two other types of treatment. We want to see how effective and safe axatilimab is at three different doses. Participants will receive one of these doses for up to two years. This is a Phase 2 study, which means it builds on earlier research to gather more information about how well the medicine works and its potential side effects in a larger group of patients.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Syndax Pharmaceuticals

Enrolment target

241

Start

04 Mar 2021

Estimated completion

01 Sep 2027

Chronic Graft-versus-host-disease

What is this study about?

This study is about a condition called chronic graft-versus-host disease, or cGVHD for short. This can happen after a stem cell transplant, which is a life-saving treatment for blood cancers and other diseases. In cGVHD, the new, healthy cells from the donor see the patient's body as 'foreign' and start to attack it. This can cause various problems in different parts of the body, making daily life difficult.

For many people, cGVHD can be managed with medicines. However, sometimes these treatments don't work well enough, or the cGVHD comes back. When this happens, doctors need new options. This study is testing a new medicine called axatilimab to see if it can help people with cGVHD that hasn't responded to at least two other treatments.

The main goals of this study are to find out if axatilimab is effective in treating cGVHD and if it's safe to use. Researchers are testing three different doses of the medicine to see which one works best with the fewest side effects. The information gathered from this study will help us understand if axatilimab could become a new treatment option for people living with cGVHD.

Key takeaways

This study is for chronic graft-versus-host disease (cGVHD) that hasn't responded to other treatments.
It's testing a new medicine called axatilimab at three different dose levels.
The goal is to find out if axatilimab is effective and safe.
Participation involves regular appointments and up to two years of treatment.
You must be at least two years old and have active cGVHD.
You can stop participating at any time.

Who may be eligible?

To join this study, you need to be at least two years old and have chronic graft-versus-host disease (cGVHD) that still needs treatment with medications to calm down your immune system. Importantly, your cGVHD must have either come back or not improved after trying at least two other types of medicine previously.

This means that your disease either got worse, new problems appeared, or your previous treatments weren't effective enough after at least three months. You might also have a combination of sudden (acute) and ongoing (chronic) GVHD symptoms, which is sometimes called 'overlap syndrome'.

Lastly, your general health and organ function should be good enough to take part in the study. Your doctor will use specific medical scales to check this, making sure you're well enough for the demands of the study.

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 at least 2 years old?
Do you have active chronic graft-vs-host disease (cGVHD)?
Has your cGVHD come back or not improved after at least two previous treatments?
Is your general health good enough to take part in a study?
Are you able to attend regular appointments for up to two years?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you would first go through a screening process to make sure it's the right fit for you. Once confirmed, you would be randomly assigned to receive one of three different doses of the new medicine, axatilimab. The medicine will be given in treatment cycles, each lasting 28 days, and you could receive treatment for up to two years.

Throughout the study, you'll have regular hospital visits for check-ups. These visits will involve blood tests, physical examinations, and other tests to see how the medicine is working and to monitor your health. Your doctors will be closely watching for any side effects and how your cGVHD symptoms are responding. After your treatment period, there will be follow-up appointments to continue monitoring your health.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. You might benefit from a new treatment for your cGVHD that hasn't worked with other medicines, and you would receive close medical attention. However, like all medicines, axatilimab might cause side effects, some of which could be serious or unexpected. The study doctors will explain all known risks to you before you decide to join. Remember, taking part is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (121)

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

University of Alabama at Birmingham - Children's of Alabama
Verified postcode
Birmingham, United States
University of Alabama at Birmingham
Verified postcode
Birmingham, United States
City of Hope
Verified postcode
Duarte, United States
University of Southern California Norris Comprehensive Cancer Center
Verified postcode
Los Angeles, United States
University of California, Los Angeles (UCLA) - Medical Center
Verified postcode
Los Angeles, United States
Stanford Cancer Center
Verified postcode
Stanford, United States
Children's National Medical Center
Verified postcode
Washington D.C., United States
University of Florida (UF)
Verified postcode
Gainesville, United States
Mayo Clinic - Jacksonville
Verified postcode
Jacksonville, United States
University of Miami
Verified postcode
Miami, United States
AdventHealth Orlando
Verified postcode
Orlando, United States
Moffitt
Verified postcode
Tampa, United States

Common questions

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

It's a complication after a stem cell transplant where the new donor cells attack the patient's body.

What is axatilimab?

It's the new medicine being tested in this study to treat cGVHD.

Why are there different doses?

Researchers want to find the dose that works best with the fewest side effects.

How long will I be in the study?

You could receive treatment for up to two years, with follow-up appointments afterwards.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time without affecting your medical care.