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Chronic Graft-versus-host-diseasePhase 3NCT06821542

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

A Phase 3 study testing INCA034176 and Best Available Therapy (BAT) in people with Chronic Graft-versus-host-disease, run by Incyte Corporation at 130 sites in 16 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

This study will be conducted to compare Axatilimab Versus Best Available Therapy in 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 12 years and older
Conditions the study is for
Chronic Graft-versus-host-disease
What is being tested?
INCA034176 and Best Available Therapy (BAT)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
130 sitesin 16 countries
Study dates
4 Jun 2025to 30 Nov 2032
Phase
Phase 3
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 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Age ≥ 12 years at the time of signing the ICF.
  • 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, pentostatin, proteasome inhibitors, imatinib, or ibrutinib.
  • 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.
  • Evidence of relapse of hematologic disease or treatment for relapse after the allo-SCT was performed, including DLI for the treatment of molecular relapse. Note: Participants who have received a scheduled DLI as part of their transplant procedure and not for management of malignancy relapse are eligible.
  • Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
  • Severe renal impairment, that is, estimated creatinine clearance < 30 mL/min measured or calculated by Cockcroft-Gault equation in adults and Schwartz formula in pediatric participants, or end-stage renal disease on dialysis.
  • Impaired liver function, defined as total bilirubin > 1.5 × ULN and/or ALT and AST > 3 × ULN in participants with no evidence of liver cGVHD.
  • History of acute or chronic pancreatitis.
Show all 9Show fewer
  • Active, symptomatic myositis.
  • Pregnant or breastfeeding.
  • Other protocol-defined Inclusion/Exclusion Criteria may apply.

Trial locations and contacts

130 sites in 16 countries.

130 sites in 16 countries

Italy31 sites

  • Ss Antonio & Biagio and C. Arrigo Hospital

    Alessandria, 15121

  • Azienda Ospedaliero Universitaria Delle Marche Clinica Ematologica

    Ancona, 60126

  • Azienda Ospedaliera Papa Giovanni Xxiii

    Bergamo, 24127

  • Azienda Ospedaliera Sant'Orsola Malpighi

    Bologna, 40138

  • Azienda Ospedaliero Universitaria Di Bologna - Policlinico S. Orsola-Malpighi - Istituto Di Ematolog

    Bologna, 40138

  • Universita Degli Studi Di Brescia - Azienda Ospedaliera Spedali Civili Di Brescia

    Brescia, 25123

  • Azienda Policlinico Vittorio Emanuele, Presidio G Rodolico, Universita Di Catania

    Catania, 95125

  • Azienda Ospedaliero Universitaria Careggi-S.O.D. Patologia Medica

    Florence, 50134

  • Irccs - Azienda Ospedaliera Universitaria - Ist San Martino

    Genova, 16132

  • Pia Fondazione Cardinale Giovanni Panico Azienda Ospedaliera

    Lecce, 73039

  • Fondazione Irccs Ca' Granda - Ospedale Maggiore Policlinico

    Milan, 20122

  • Istituto Di Ricovero E Cura A Carattere Scientifico (Irccs) - Ospedale San Raffaele (Hsr) (Istituto

    Milan, 20132

  • Asst Grande Ospedale Metropolitano Niguarda

    Milan, 20162

  • Azienda Ospedaliera San Gerardo Di Monza

    Monza, 20900

  • Azienda Ospedaliera Di Rilievo Nazionale (Aorn) 'Antonio Cardarelli'

    Naples, 80131

  • Azienda Ospedaliero Universitaria Federico Ii Di Napoli

    Naples, 80131

  • Azienda Ospedale Universita Di Padova

    Padova, 35128

  • Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello

    Palermo, 90146

  • Irccs Fondazione Policlinico San Matteo

    Pavia, 27100

  • Presidio Ospedaliero Spirito Santo Azienda U.S.L. Pescara

    Pescara, 65124

  • Morelli Hospital

    Reggio Calabria, 89133

  • Aou Policlinico Umberto I

    Roma, 00161

  • A. Gemelli University Hospital, Catholic University of the Sacred Heart

    Roma, 00168

  • Fondazione Ptv Policlinico Tor Vergata

    Rome, 00133

  • Irccs Ospedale Pediatrico Bambino Gesu

    Rome, 00165

  • Irccs Istituto Clinico Humanitas

    Rozzano, 20089

  • Fondazione Ircss Casa Sollievo Della Sofferenza

    San Giovanni Rotondo, 71013

  • A.O.U. Citta Della Salute E Della Scienza Di Torino

    Torino, 10126

  • Ospedale Santa Maria Di Ca' Foncello

    Treviso, 31100

  • Clinica Ematologica, Dism Azienda Ospedaliero Universitaria

    Udine, 33100

  • Uoc Ematologia E Centro Trapianti Midollo Osseo Br, Ospedale Borgo Roma

    Verona, 37134

Other trials in Italy

Spain21 sites

  • Institut Catala D'Oncologia Badalona, Hospital Germans Trias I Pujol

    Badalona, 08916

  • Hospital Universitario Vall D'Hebron

    Barcelona, 08035

  • Hospital Clinic de Barcelona (Hospital Clinic I Provincial)

    Barcelona, 08036

  • Hospital de La Santa Creu I Sant Pau

    Barcelona, 08041

  • Institut Catala D Oncologia

    Barcelona, 08908

  • Hospital Clinico Universitario Virgen de La Arrixaca

    El Palmar, 30120

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

    Esplugues de Llobregat, 08950

  • Hospital Universitario Virgen de Las Nieves

    Granada, 18014

  • Hospital Universitario de Gran Canaria Doctor Negrín

    Las Palmas de Gran Canaria, 35010

  • Hospital General Universitario Gregorio Maranon (Hgugm)

    Madrid, 28007

  • Hospital Universitario Infantil Nino Jesus

    Madrid, 28009

  • Hospital Universitario Ramon Y Cajal

    Madrid, 28034

  • Hospital Universitario 12 de Octubre

    Madrid, 28041

  • Hospital Regional Universitario de Malaga

    Málaga, 29010

  • Hospital Clinico Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario de Donostia

    SAN Sebasttian, 20014

  • Hospital Universitario Marques de Valdecilla

    Santander, 39008

  • Hospital Universitario Virgen Del Rocio

    Seville, 41013

  • Hospital Clinico Universitario de Valencia (Instituto de Investigacion Sanitaria Incliva)

    Valencia, 46010

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

    Valencia, 46026

  • Hospital Universitario Miguel Servet

    Zaragoza, 50009

Other trials in Spain

Germany17 sites

  • Uniklinik Rwth Aachen Medizinische Klinik Iv

    Aachen, 52074

  • Charite - Universitaetsmedizin Berlin

    Berlin, 10117

  • Universitaetsklinikum Koeln

    Cologne, 50937

  • University Hospital Carl Gustav Carus

    Dresden, 01307

  • Universitatsklinikum Erlangen

    Erlangen, 91054

  • Klinikum Der Johann Wolfgang Goethe-Universitaet

    Frankfurt am Main, 60590

  • Universitaetsklinikum Freiburg

    Freiburg im Breisgau, 79106

  • Universitaetsmedizin Goettingen

    Göttingen, 37075

  • Universitaetsklinikum Halle

    Halle, 06120

  • Universitaetsklinikum Des Saarlandes

    Homburg, 66421

  • Universitaetsklinikum Schleswig-Holstein, Uksh-Campus Kiel

    Kiel, 24105

  • University Hospital Leipzig

    Leipzig, 04103

  • Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii

    Mainz, 55131

  • Universitaetsklinikum Muenster

    Münster, 48149

  • University Clinic Regensburg

    Regensburg, 93053

  • University Hospital of Ulm, Universitatsklinikum Ulm

    Ulm, 89081

  • University of Wuerzburg

    Würzburg, 97080

Other trials in Germany

France14 sites

  • Chu D'Amiens-Picardie - Hopital Sud

    Amiens, 80054

  • CHU CAEN

    Caen, 14000

  • Centre Hospitalier Regional Universitaire (Chru) de Lille - Hopital Claude Huriez

    Lille, 59037

  • Institut Paoli Calmettes

    Marseille, 13009

  • Chu de Nantes

    Nantes, 44000

  • Centre Hospitalier Universitaire de Nice,Hopital L Archet

    Nice, 06200

  • Hospital Saint-Louis - Aphp

    Paris, 75010

  • Hopital Universitaire Robert Debre

    Paris, 75019

  • Centre Hospitalier Universitaire de Bordeaux - Hopital Haut-Leveque

    Pessac, 33600

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, 69495

  • Centre Henri Becquerel

    Rouen, 76038

  • Institut Claudius Regaud Cancer Comprehensive Center - Iuct Oncopole

    Toulouse, 31059

  • Chru Nancy

    Vandœuvre-lès-Nancy, 54511

  • Gustave Roussy Cancer Campus Grand Paris- (Institut de Cancerologie Gustave-Roussy)

    Villejuif, 94805

Other trials in France

Belgium6 sites

  • Az Sint-Jan Brugge Av

    Bruges, 08000

  • Chu Ucl Namur Site Godinne

    Godinne-mont, 05530

  • Universitair Ziekenhuis Leuven

    Leuven, 03000

  • Chu Liege -Centre Hospitalier Universitaire Sart Tilman

    Liège, 04000

  • Algemeen Ziekenhuis Delta

    Roeselare, 08800

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

    Woluwe-Saint-Lambert, 01200

Other trials in Belgium

Netherlands6 sites

  • Amsterdam University Medical Center (Amsterdam Umc), Vrije University Medical Center (Vumc)

    Amsterdam, 1081 HV

  • University Medical Center Groningen

    Groningen, 9713 GZ

  • Leiden University Medical Center (Leids Universitair Medisch Centrum (Lumc))

    Leiden, 2333 ZA

  • Maastricht University Medical Center (Mumc)

    Maastricht, 6229 HX

  • Erasmus Medisch Centrum 1

    Rotterdam, 3015 GD

  • Universitair Medisch Centrum Utrecht (Umc Utrecht)

    Utrecht, 3584 CX

Other trials in Netherlands

Austria5 sites

  • Medical University of Graz

    Graz, 08036

  • Medizinische Universitaet Innsbruck - Universitaetsklinik Fuer Innere Medizin Iii

    Innsbruck, 06020

  • Krankenhaus Der Elisabethinen Linz Gmbh

    Linz, 04020

  • Medizinische Universitat Wien, Universitatsklinik Fur Innere Medizin I

    Vienna, 01090

  • St. Anna Childrens Hospital

    Vienna, 01090

Other trials in Austria

Sweden5 sites

  • Sahlgrenska University Hospital

    Gothenburg, 413 45

  • University of Gothenburg and Queen Silvia Children S Hospital At Sahlgrenska University Hospital

    Gothenburg, 41650

  • Skanes Universitetssjukhus - Universitetssjukhuset I Lund

    Lund, 221 85

  • Karolinska Universitetssjukhuset, Centrum For Cellterapi Och Allogen Stamcellstransplantation

    Stockholm, 14186

  • Uppsala University Hospital

    Uppsala, 75185

Other trials in Sweden

United Kingdom5 sites

  • Cardiff and Vale Nhs Trust - University Hospital of Wales (Uhw)

    Cardiff, CF14 4XW

  • Queen Elizabeth University Hospital

    Glasgow, G51 4TF

  • St James'S University Hospital - Leeds Teaching Hospitals Nhs Trust

    Leeds, LS9 7TF

  • University Hospitals of Leicester Nhs Trust-Leicester Royal Infirmary

    Leicester, LE1 5WW

  • King'S College Hospital Nhs Foundation Trust

    London, SE5 9RS

Other trials in United Kingdom

Greece4 sites

  • General Hospital of Athens Evangelismos - Eye Hospital

    Athens, 10676

  • Attikon Hospital and Cutaneous Lymphoma Clinic-Athens University Medical School

    Athens, 12462

  • University Hospital of Patras

    RIO Patras, 26504

  • General Hospital of Thessalonikis George Papanikolaou - Gene and Cell Therapy Center

    Thessaloniki, 57010

Other trials in Greece

Switzerland4 sites

  • University Hospital Basel

    Basel, 04031

  • Hopitaux Universitaires de Geneve (Hug) - Centre de Recherche Clinique (Crc)

    Geneva, 01205

  • Universitatsspital Zurich

    Zurich, 08091

  • Universitats-Kinderspital Zurich Eleonorenstiftung

    Zurich, CH-8032

Other trials in Switzerland

Czechia3 sites

  • Fakultni Nemocnice Hradec Kralove

    Hradec Králové, 500 05

  • Fakultni Nemocnice Ostrava

    Ostrava-poruba, 708 52

  • Ustav Hematologie A Krevni Transfuze (Uhkt), the Institute of Hematology and Blood Transfusion (Ihbt

    Prague, 12808

Other trials in Czechia

Finland3 sites

  • Helsinki University Central Hospital

    Helsinki, 00029

  • New Children'S Hospital, University of Helsinki and Helsinki University Hospital

    Helsinki, 00290

  • Turku University Hospital

    Turku, 20520

Other trials in Finland

Portugal3 sites

  • Ipo Lisboa Francisco Gentil

    Lisbon, 1099-035

  • Centro Hospitalar Universitario Lisboa Norte Epe

    Lisbon, 1649-028

  • Instituto Portugues de Oncologia Do Porto Francisco Gentil E.P.E

    Porto, 4200-072

Other trials in Portugal

Norway2 sites

  • Haukeland University Hospital

    Bergen, 05021

  • Rikshospitalet University Hospital

    Oslo, 00027

Other trials in Norway

Ireland1 site

  • St. James Hospital

    Dublin, D08 NHY1

Other trials in Ireland

About this study

Official title: A Phase 3, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy

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

Being tested
INCA034176, Best Available Therapy (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

    IV infusion

  • Best Available Therapy (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 NCT06821542
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 Therapy (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 for each treatment group 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

Secondary outcomes (12)

  1. Failure-free survival (FFS)

    Defined as the time from the date of randomization to the date of addition or initiation of another systemic therapy for cGVHD, relapse of underlying disease, or death due to any cause.

    Measured: Up to 5 years

  2. Proportion of participants with a ≥ 7-point improvement in modified Lee Symptom Scale (mLSS) total score

    Measured: Up to 5 years

  3. Overall Response at 12 months

    Defined as CR or PR at 12 months in the absence of new systemic therapy for cGVHD.

    Measured: 12 months

  4. Best Overall Response (BOR)

    Defined as the best response of CR or PR in the first 6 months (up to and including Cycle 7 Day 1), and at any timepoint up to the initiation of new therapy for cGVHD.

    Measured: Up to 5 years

  5. DOR (in responders only)

    Defined as the time from the date of first response (PR or CR) to the date of progression of cGVHD from baseline scoring, start 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

  6. Organ-specific response

    Organ-specific response as defined in the protocol.

    Measured: Up to 5 years

  7. Overall Survival (OS)

    Defined as the time from the date of randomization to the date of death due to any cause.

    Measured: Up to 5 years

  8. Nonrelapse mortality (NRM)

    Defined as the time from the date of randomization to the date of death not preceded by relapse of primary hematologic disease.

    Measured: Up to 5 years

  9. Time to primary hematologic disease relapse

    Defined as the time from the date of randomization to the date of relapse.

    Measured: Up to 5 years

  10. Percent reduction in daily corticosteroid dose at 6 months

    Measured: 6 months

  11. Proportion of participants who tapered off all corticosteroids at 6 months

    Measured: 6 months

  12. 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 posted12 Feb 2025
  2. Study start4 Jun 2025
  3. Primary completion31 May 2028 (estimated)
  4. Study completion30 Nov 2032 (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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06821542
Sponsor's protocol ID
INCA034176-355
Other IDs
2024-518973-32-00
Status last verified
Aug 2026
First submitted
6 Feb 2025
Last updated
25 Aug 2026
Results posted
No
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 3 study testing INCA034176 and Best Available Therapy (BAT) in people with Chronic Graft-versus-host-disease, run by Incyte Corporation at 130 sites in 16 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: under way, not looking for participants, as of the sponsor's last update on 25 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 130 sites in Italy, Spain, Germany, France, Belgium and 11 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Objective Response (OR) at 6 months (time frame: 6 months). 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 NCT06821542.

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 25 Aug 2026.

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