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New hope for kids with chronic Graft-Versus-Host disease: axatilimab trial launches

NCT ID NCT07124078

Disease control Sponsor: Incyte Corporation Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Sep 16, 2026 · Updated 5 times

Summary

This study tests a drug called axatilimab against the best available treatments for children aged 2 to 18 with chronic graft-versus-host disease (cGVHD), a complication after a stem cell transplant. The trial enrolls 60 participants who have already tried at least two other treatments. The goal is to see if axatilimab can improve symptoms better than current options.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
axatilimab
What this could lead to
If it works, this could offer a new treatment option for children with chronic graft-versus-host disease who have not responded to other therapies.
What could go wrong
This is a mid-stage trial with only 60 participants, so results may not apply to all patients. The drug may not work better than existing options, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Jul 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Aged 2 to \< 18 years at the time of randomization. * Active, moderate to severe cGVHD, requiring systemic immune suppression. * Participants with refractory or recurrent cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib. * Concomitant use of systemic corticosteroids is allowed. Participants on systemic corticosteroids must be on a stable dose of corticosteroids for at least 2 weeks prior to C1D1. Topical and inhaled corticosteroid agents are allowed. * Participants must accept to be treated with one of the following BAT options on C1D1: CNI (cyclosporine or tacrolimus), ECP, MMF, an mTOR inhibitor (everolimus or sirolimus), rituximab, imatinib, methotrexate, ibrutinib, or pentostatin. * History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood). Recipients of myeloablative, nonmyeloablative, or reduced-intensity conditioning are eligible. Exclusion Criteria: * Receipt of more than 1 prior allo-HCT. Prior autologous HCT is allowed, including autologous CAR T-cell therapy given before the allo-SCT. * Documented evidence of relapse of the primary hematologic disease or receipt of treatment for relapse after allo-SCT, including DLI for treatment of any malignancy relapse, including molecular relapse. Autologous and allogeneic donor-derived CAR T-cell therapy after allo-SCT are not allowed. Note: Participants who received DLI solely for the management of mixed chimerism or as part of the planned transplant procedure and not for treatment of malignancy relapse (including molecular relapse), are eligible. * Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1. * Severe renal impairment, that is, GFR \< 30 mL/min/1.73 m2 as estimated using modified Schwartz formula, or end-stage renal disease on dialysis. * Impaired liver function, defined as total bilirubin \> 1.5 × ULN or ALT \> 3 × ULN or AST \> 3 × ULN in participants with no evidence of liver cGVHD. * History of acute or chronic pancreatitis. * Active, symptomatic myositis. * Female adolescent participants who are pregnant or breastfeeding. Other protocol-defined Inclusion/Exclusion Criteria may apply.

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Conditions

The condition(s) this trial relates to.

Bronchiolitis Obliterans Syndrome chronic graft versus host disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Chronic graft versus host disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    41 sites in 6 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Addenbrooke'S Hospital - Cambridge University Hospitals Nhs Foundation Trust

    RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Adventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant

    NOT_YET_RECRUITING

    Orlando, Florida, 32804, United States

  • Azienda Ospedaliera Universitaria Di Padova

    RECRUITING

    Padua, 35128, Italy

  • Birmingham Children'S Hospital - Birmingham Women'S and Children'S Nhs Foundation Trust

    RECRUITING

    Birmingham, B4 6NH, United Kingdom

  • Charite Campus Virchow

    RECRUITING

    Berlin, 13353, Germany

  • Children'S Hospital Los Angeles Specialt

    NOT_YET_RECRUITING

    Los Angeles, California, 90027, United States

  • Childrens Healthcare of Atlanta

    RECRUITING

    Atlanta, Georgia, 30329, United States

  • Childrens Hospital of Pittsburgh

    NOT_YET_RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • Childrens National Medical Center

    RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • City of Hope Medical Center

    RECRUITING

    Duarte, California, 91010, United States

  • Duke Cancer Center

    NOT_YET_RECRUITING

    Durham, North Carolina, 27710, United States

  • Fondazione Irccs Policlinico San Matteo Di Pavia Oncoematologia Pediatrica, Universita Di Pavia

    RECRUITING

    Pavia, 27100, Italy

  • Fondazione Irccs San Gerardo Dei Tintori

    RECRUITING

    Monza, 20900, Italy

  • Fred Hutchinson Cancer Research Center

    RECRUITING

    Seattle, Washington, 98109, United States

  • Great Ormond Street Hospital Nhs Trust

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hospital Infantil Unversitario Nino Jesus

    NOT_YET_RECRUITING

    Madrid, 28009, Spain

  • Hospital Regional Universitario de Malaga- Hospital Materno Infantil

    RECRUITING

    Málaga, 29011, Spain

  • Hospital Sant Joan de Deu Barcelona - Children'S Hospital

    NOT_YET_RECRUITING

    Esplugues de Llobregat, 08950, Spain

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, 28046, Spain

  • Hospital Universitario Vall D'Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • Hospital Universitario Virgen de La Arrixaca

    RECRUITING

    Murcia, 30120, Spain

  • Hôpital Universitaire Des Enfants Reine Fabiola

    RECRUITING

    Brussels, Belgium

  • Irccs - Aou Di Bologna - Sant'Orsola Malpighi

    RECRUITING

    Bologna, 40138, Italy

  • Irccs Istituto G. Gaslini, Universita Di Genova

    RECRUITING

    Genova, 16147, Italy

  • Irccs Ospedale Pediatrico Bambino Gesu

    RECRUITING

    Rome, 00165, Italy

  • Klinikum Der Johann Wolfgang Goethe-Universitaet

    RECRUITING

    Frankfurt am Main, 60590, Germany

  • Leeds Childrens Hospital

    RECRUITING

    Leeds, LS1 3EX, United Kingdom

  • Levine Cancer Institute

    RECRUITING

    Charlotte, North Carolina, 28203, United States

  • Lucile Packard Child Hospital End

    RECRUITING

    Palo Alto, California, 94304, United States

  • Medizinische Hochschule Hannover (Mhh) - Zentrum Fuer Kinderheilkunde Und Jugendmedizin - Klinik Fue

    NOT_YET_RECRUITING

    Hanover, 30625, Germany

  • Memorial Sloan Kettering Cancer Center

    RECRUITING

    New York, New York, 10065, United States

  • Royal Manchester Children'S Hospital - Manchester University Nhs Foundation Trust

    RECRUITING

    Manchester, M139WL, United Kingdom

  • Royal Victoria Infirmary - the Newcastle Upon Tyne Hospitals Nhs Foundation Trust

    RECRUITING

    Newcastle, NE1 4LP, United Kingdom

  • Sheffield Children'S Hospital - Sheffield Children'S Nhs Foundation Trust

    RECRUITING

    Sheffield, S10 2TH, United Kingdom

  • St Mary'S Hospital, Paddington - Imperial College Healthcare Nhs Trust

    RECRUITING

    London, W2 1NY, United Kingdom

  • The Royal Hospital For Children - Glasgow Health Board

    RECRUITING

    Glasgow, G51 4TF, United Kingdom

  • Universitaetsklinikum Freiburg Kinder- Und Jugendklinik (Kjk)

    RECRUITING

    Freiburg im Breisgau, 79106, Germany

  • Universitaetsklinikum Hamburg-Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

  • Universitat de Valencia - Hospital Universitari I Politecnic La Fe de Valencia (Hospital La Fe Bulev

    RECRUITING

    Valencia, 46026, Spain

  • Universite Catholique de Louvain (Ucl) - Cliniques Universitaires Saint-Luc

    RECRUITING

    Woluwe-Saint-Lambert, 01200, Belgium

  • University Hospital Regensburg

    NOT_YET_RECRUITING

    Regensburg, 93053, Germany

More trials for these conditions

Other studies related to the condition(s) this trial covers.