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Chronic Graft-versus-host-diseasePhase 2NCT07124078

A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

A Phase 2 study testing INCA034176 and Best available Treatment (BAT) in people with Chronic Graft-versus-host-disease, run by Incyte Corporation at 41 sites in 6 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy. Read more about this study

Who is it for?
Ages 2 to 17 years
Conditions the study is for
Chronic Graft-versus-host-disease
What is being tested?
INCA034176 and Best available Treatment (BAT)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
41 sitesin 6 countries
Study dates
20 May 2026to 31 Jul 2029
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 9Show fewer
  • Active, symptomatic myositis.
  • Female adolescent participants who are pregnant or breastfeeding.
  • Other protocol-defined Inclusion/Exclusion Criteria may apply.

Trial locations and contacts

41 sites in 6 countries. Contact the study team or a site near you to ask about taking part.

Incyte Corporation Call Center (US) contact

1.855.463.3463

medinfo@incyte.com

Incyte Corporation Call Center (ex-US) contact

+800 00027423

eumedinfo@incyte.com

41 sites in 6 countries

United States11 sites, 11 recruiting

  • Childrens Healthcare of Atlanta

    Atlanta, Georgia, 30329

    Recruiting

  • Levine Cancer Institute

    Charlotte, North Carolina, 28203

    Recruiting

  • City of Hope Medical Center

    Duarte, California, 91010

    Recruiting

  • Duke Cancer Center

    Durham, North Carolina, 27710

    Not yet recruiting

  • Children'S Hospital Los Angeles Specialt

    Los Angeles, California, 90027

    Not yet recruiting

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

    Recruiting

  • Adventhealth Pediatric Cellular Therapy and Blood and Marrow Transplant

    Orlando, Florida, 32804

    Not yet recruiting

  • Lucile Packard Child Hospital End

    Palo Alto, California, 94304

    Recruiting

  • Childrens Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

    Not yet recruiting

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109

    Recruiting

  • Childrens National Medical Center

    Washington D.C., District of Columbia, 20010

    Recruiting

Other trials in United States

United Kingdom9 sites, 9 recruiting

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

    Birmingham, B4 6NH

    Recruiting

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

    Cambridge, CB2 0QQ

    Recruiting

  • The Royal Hospital For Children - Glasgow Health Board

    Glasgow, G51 4TF

    Recruiting

  • Leeds Childrens Hospital

    Leeds, LS1 3EX

    Recruiting

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

    London, W2 1NY

    Recruiting

  • Great Ormond Street Hospital Nhs Trust

    London, WC1N 3JH

    Recruiting

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

    Manchester, M139WL

    Recruiting

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

    Newcastle, NE1 4LP

    Recruiting

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

    Sheffield, S10 2TH

    Recruiting

Other trials in United Kingdom

Spain7 sites, 7 recruiting

  • Hospital Universitario Vall D'Hebron

    Barcelona, 08035

    Recruiting

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

    Esplugues de Llobregat, 08950

    Not yet recruiting

  • Hospital Infantil Unversitario Nino Jesus

    Madrid, 28009

    Not yet recruiting

  • Hospital Universitario La Paz

    Madrid, 28046

    Recruiting

  • Hospital Regional Universitario de Malaga- Hospital Materno Infantil

    Málaga, 29011

    Recruiting

  • Hospital Universitario Virgen de La Arrixaca

    Murcia, 30120

    Recruiting

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

    Valencia, 46026

    Recruiting

Other trials in Spain

Germany6 sites, 6 recruiting

  • Charite Campus Virchow

    Berlin, 13353

    Recruiting

  • Klinikum Der Johann Wolfgang Goethe-Universitaet

    Frankfurt am Main, 60590

    Recruiting

  • Universitaetsklinikum Freiburg Kinder- Und Jugendklinik (Kjk)

    Freiburg im Breisgau, 79106

    Recruiting

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246

    Recruiting

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

    Hanover, 30625

    Recruiting

  • University Hospital Regensburg

    Regensburg, 93053

    Not yet recruiting

Other trials in Germany

Italy6 sites, 6 recruiting

  • Irccs - Aou Di Bologna - Sant'Orsola Malpighi

    Bologna, 40138

    Recruiting

  • Irccs Istituto G. Gaslini, Universita Di Genova

    Genova, 16147

    Recruiting

  • Fondazione Irccs San Gerardo Dei Tintori

    Monza, 20900

    Recruiting

  • Azienda Ospedaliera Universitaria Di Padova

    Padua, 35128

    Recruiting

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

    Pavia, 27100

    Recruiting

  • Irccs Ospedale Pediatrico Bambino Gesu

    Rome, 00165

    Recruiting

Other trials in Italy

Belgium2 sites, 2 recruiting

  • Hôpital Universitaire Des Enfants Reine Fabiola

    Brussels

    Recruiting

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

    Woluwe-Saint-Lambert, 01200

    Recruiting

Other trials in Belgium

About this study

Official title: A Phase 2, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)

This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.

Being tested
INCA034176, Best available Treatment (BAT)
Keywords
cGVHD
Medical subject headings
Bronchiolitis Obliterans Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • INCA034176Drug

    Also called Axatilimab

    Axatilimab at the protocol-defined dose.

  • Best available Treatment (BAT)Drug

    Also called BAT could include:, • Calcineurin inhibitor (cyclosporine or tacrolimus), • Extracorporeal photopheresis (ECP), • Mycophenolate mofetil (MMF)

    Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.

Study groups in NCT07124078
Study groupRoleWhat they receive
AxatilimabExperimentalAxatilimab at the protocol-defined dose.
Receives: Drug: INCA034176
Best available Treatment (BAT)ExperimentalBest Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Receives: Drug: Best available Treatment (BAT)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Objective Response (OR) at 6 months

    Defined as complete response (CR) or partial response (PR) at 6 months (Cycle 7 Day 1, 28-day cycles) in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.

    Measured: 6 months

  2. United States (US): Best overall Response (BOR)

    Defined as a best response of CR or PR in the first 6 months (up to and including Cycle 7 Day 1) in the absence of new systemic therapy for cGVHD.

    Measured: Up to 6 months

Secondary outcomes (17)

  1. Pharmacokinetics Parameter (PK): Cmax of axatilimab

    Defined as maximum observed plasma concentration of axatilimab.

    Measured: Up to 5 years

  2. Pharmacokinetics Parameter: Tmax of axatilimab

    Defined as the time to reach the maximum plasma concentration of axatilimab.

    Measured: Up to 5 years

  3. Pharmacokinetics Parameter: Cmin of axatilimab

    Defined as the minimum observed plasma concentration of axatilimab.

    Measured: Up to 5 years

  4. Pharmacokinetics Parameter: AUC(0-t) of axatilimab

    Defined as the area under the concentration-time curve up to the last measurable concentration of axatilimab.

    Measured: Up to 5 years

  5. Pharmacokinetics Parameter: AUC 0-∞ of axatilimab

    Defined as the area under the concentration-time curve from 0 to infinity of axatilimab.

    Measured: Up to 5 years

  6. Pharmacokinetics Parameter: CL of axatilimab

    Defined as the apparent oral dose clearance of axatilimab.

    Measured: Up to 5 years

  7. Pharmacokinetics Parameter: Vz of axatilimab

    Defined as the apparent oral dose volume of distribution of axatilimab.

    Measured: Up to 5 years

  8. Pharmacokinetics Parameter: t1/2 of axatilimab

    Defined as the apparent terminal phase disposition half-life of axatilimab.

    Measured: Up to 5 years

  9. Best Overall Response (BOR) on study

    Defined as a best response of CR or PR at any timepoint up to the initiation of new systemic therapy for cGVHD.

    Measured: Up to 5 years

  10. BOR in the first 6 months

    Defined as a best response of CR or PR in the first 6 months (up to and including Cyle 7 Day 1) in the absence of new systemic therapy for cGVHD.

    Measured: Up to 6 months

  11. Overall Response (OR) at 12 months

    Defined as CR or PR at 12 months (Cycle 13 Day 1) in the absence of new systemic therapy for cGVHD.

    Measured: 12 months

  12. US: OR at 6 months

    Defined as CR or PR at 6 months (Cycle 7 Day 1) in the absence of new systemic therapy for cGVHD.

    Measured: 6 months

  13. Duration of Response (DOR) (in responders only)

    Defined as the time from the date of first response (PR or CR) to the date of progression of cGVHD, initiation of new systemic treatment for cGVHD, or death from any cause, whichever comes first. An additional measure of response durability will consider DOR as the time from the date of first response to the date of new systemic therapy for cGVHD or death from any cause, whichever occurs first.

    Measured: Up to 5 years

  14. Organ-specific response

    Organ-specific response as defined in the protocol.

    Measured: Up to 5 years

  15. Percent reduction in daily corticosteroid dose at 6 months

    Defined as Percent reduction in daily corticosteroid dose at Cycle 7 Day 1, and participants successfully tapered off all corticosteroids at Cycle 7 Day 1.

    Measured: 6 months

  16. Changes in parameters collected using the pediatric stem cell quality of life (Q0L) questionnaire (PedsQL Stem Cell Transplant Module)

    Measured: Up to 5 years

  17. Number of participants with Treatment-emergent Adverse Events (TEAEs)

    Defined as adverse events reported for the first time or worsening of a pre-existing event after the first dose of study treatment.

    Measured: Up to 5 years and 30 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Aug 2025
  2. Study start20 May 2026
  3. Primary completion31 Jan 2029 (estimated)
  4. Study completion31 Jul 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
60 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07124078
Sponsor's protocol ID
INCA034176-256
Status last verified
Oct 2026
First submitted
8 Aug 2025
Last updated
9 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Incyte shares data with qualified external researchers after a research proposal is submitted. These requests are reviewed and approved by a review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. The trial data availability is according to the criteria and process described on https://www.incyte.com/our-company/compliance-and-transparency

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing INCA034176 and Best available Treatment (BAT) in people with Chronic Graft-versus-host-disease, run by Incyte Corporation at 41 sites in 6 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 41 sites in United States, United Kingdom, Spain, Germany, Italy and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Incyte Corporation Call Center (US) (1.855.463.3463, medinfo@incyte.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Objective Response (OR) at 6 months (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07124078.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2026.

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Clinical Trial NCT07124078 | AzTrials