Study of Axatilimab compared to standard therapy in children with chronic graft-versus-host disease who have previously received at least 2 treatments

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What is this study about?

This study focuses on Chronic Graft-Versus-Host Disease, a condition that can occur after a stem cell transplant when the donated cells attack the recipient’s body. The study will evaluate a new medication called axatilimab, given as an intravenous solution, compared to currently available treatments in children and teenagers who have already tried at least two other treatments that didn’t work well enough.

The purpose of this research is to determine how well axatilimab works compared to other standard treatments in managing chronic graft-versus-host disease. During the study, participants will receive either axatilimab or one of several commonly used treatments for this condition. The treatment period may continue for up to 24 months.

Throughout the study, doctors will monitor how participants respond to treatment, including changes in their symptoms and overall health. They will also track how well participants can reduce their use of corticosteroids, which are commonly used to treat this condition. The study will collect information about quality of life and check for any side effects of the treatment.

1 Initial assessment

Your eligibility for the study will be evaluated based on specific criteria, including age (between 2 and 18 years) and having chronic Graft-Versus-Host Disease that requires systemic treatment.

Your medical history will be reviewed to confirm you have received at least 2 previous treatments, including corticosteroids and ruxolitinib.

Your performance status and blood count levels will be checked to ensure they meet the required criteria.

2 Treatment assignment

You will be randomly assigned to receive either axatilimab or one of the available therapy options.

If assigned to axatilimab, you will receive it through intravenous infusion.

If assigned to other therapy, you will receive one of the following treatments: cyclosporine, tacrolimus, ECP, MMF, everolimus, sirolimus, rituximab, imatinib, methotrexate, or ibrutinib.

3 Treatment period

The treatment will continue for at least 6 months.

If you are currently taking corticosteroids, you may continue them at a stable dose.

Regular assessments will monitor your response to treatment.

Your quality of life will be evaluated using a special questionnaire for stem cell transplant patients.

4 Monitoring and assessment

Your response to treatment will be evaluated at 6 months and 12 months.

Regular safety assessments will include blood tests and clinical examinations.

Your corticosteroid dose may be adjusted based on your response to treatment.

Any side effects or changes in your condition will be monitored and recorded.

5 Study completion

The study is expected to continue until January 2029.

Your participation will be completed after the planned treatment period or if you need to start a new treatment for your condition.

Final assessments will be conducted to evaluate the overall response to treatment.

Who Can Join the Study?

Age between 2 and 17 years old at the time of joining the study
Must be able to understand and willing to sign an informed consent form (for children, parents/guardians must provide consent, and where applicable, children should sign an assent form)
Have active moderate to severe chronic Graft-Versus-Host Disease that requires systemic treatment (medicines that affect the entire body)
Previous history of allogeneic stem cell transplant from any donor type using bone marrow, blood stem cells, or cord blood
Must have received at least 2 previous treatments, including corticosteroids and ruxolitinib, that did not work well enough
Must have a performance score of 60 or higher on either the Karnofsky scale (for those 16 years or older) or Lansky scale (for those under 16 years), which measure ability to perform daily activities
Must have adequate blood cell counts:
– White blood cells (neutrophils) at least 0.5 × 109/L
– Platelets at least 20 × 109/L
Can be taking corticosteroids, but must be on a stable dose for at least 2 weeks before starting the study
Must be willing to accept one of the following treatment options: cyclosporine, tacrolimus, ECP, MMF, everolimus, sirolimus, rituximab, imatinib, methotrexate, or ibrutinib
Must agree to avoid pregnancy or fathering children during the study

Who Cannot Join the Study?

Active or uncontrolled infection requiring systemic treatment
History of HIV, active hepatitis B, or active hepatitis C infection
Active bleeding or high risk of bleeding complications
Pregnant or breastfeeding women
Uncontrolled or severe heart conditions
Major surgery within 4 weeks before starting the study
Participation in other clinical trials within 30 days
Current or recent use of other investigational drugs
Severe liver problems (liver function tests more than 3 times above normal)
Severe kidney problems (requiring dialysis)
Active or untreated cancer other than successfully treated non-melanoma skin cancer
Mental conditions that could interfere with following study procedures
History of severe allergic reactions to similar medications
Unstable medical conditions that could affect study participation
Use of medications that could interact with the study drug

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Medizinische Hochschule Hannover	Hanover	Germany
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Universitaet Leipzig	Leipzig	Germany

Other Sites

Site Name	City	Country
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Fondazione IRCCS San Gerardo Dei Tintori	Monza	Italy
Azienda Ospedaliera di Padova	Padua	Italy
Universitaetsklinikum Regensburg AöR	Regensburg	Germany
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
University Clinical Hospital Virgen De La Arrixaca	Murcia	Spain
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
University Childrens Hospital Queen Fabiola	Brussels	Belgium
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Catleibgc Utallwhhdcwfzk Sphjsjmas	Woluwe-Saint-Lambert	Belgium
Utilmuuvay Mmdchuy Ctsusi Hpyztyibtioaqfjqh	Hamburg	Germany
Gxnquq Uguqhlsbqe Fwjxmonnj	Frankfurt	Germany
Adlcpvx Urctw Sstiscwjp Lplbhw Dz Bdxbbcd	Bologna	Italy
Fzmlamppo Piup Lb Imqegkunxgfqt Bkmdyxdrx Diw Heunhhvm Uekxnzvdooqtq Lq Pdu	Madrid	Spain
Hvtxffvy Veui dlnhzyjk	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	30.09.2025
Germany	Recruiting	30.09.2025
Italy	Recruiting	30.09.2025
Spain	Recruiting	30.09.2025

Trial locations

Investigated drugs:

Axatilimab

Axatilimab is a medication being studied for treating chronic graft versus host disease (cGVHD) in children. This condition can occur after stem cell transplantation when the donated cells attack the recipient’s body. The medication works by targeting specific immune system cells that contribute to the development of cGVHD. It is designed to help reduce the symptoms and severity of the condition in patients who have already tried at least two other treatments.

Best Available Therapy (BAT) refers to the current standard treatments that doctors typically use for managing chronic graft versus host disease. This may include various medications that are already approved and commonly used to treat this condition. The specific treatment chosen depends on the individual patient’s needs and medical history.

Investigated diseases:

Chronic graft versus host disease

Chronic Graft-Versus-Host Disease – A condition that occurs after bone marrow or stem cell transplantation where the donated cells attack the recipient’s body tissues. The disease typically develops gradually and can affect multiple organs including the skin, eyes, mouth, liver, and lungs. It usually appears more than 100 days after transplantation and can persist for months or years. The condition causes inflammation and fibrosis in various parts of the body, leading to symptoms such as skin changes, dry mouth, and eye irritation. The severity can range from mild to severe, affecting a person’s daily activities and quality of life.

Trial ID:

2025-521849-25-00

Protocol code:

INCA034176-256

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of Axatilimab compared to standard therapy in children with chronic graft-versus-host disease who have previously received at least 2 treatments

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?