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AzTrials

Chronic Graft-versus-host-diseasePhase 2NCT06388564

A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

A Phase 2 study testing Axatilimab, Ruxolitinib and Corticosteroids in people with Chronic Graft-versus-host-disease, run by Incyte Corporation at 68 sites in 7 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 determine the preliminary efficacy of axatilimab in combination with ruxolitinib and to assess the contribution of axatilimab to the combination treatment effect in participants with cGVHD. 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?
Axatilimab, Ruxolitinib and Corticosteroids
Could I receive a placebo?
No, everyone receives an active treatment
How many people
130 participants
Where
68 sitesin 7 countries
Study dates
11 Oct 2024to 30 Mar 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 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • ≥ 12 years of age at the time of informed consent.
  • New-onset moderate or severe cGVHD, as defined by the 2014 NIH Consensus Development Project Criteria for Clinical Trials in cGVHD, requiring systemic therapy.
  • History of 1 allo-SCT (any type of stem cell donor, any conditioning regimen, and source of hematopoietic stem cells).
  • Adequate hematologic function independent of platelet transfusion and growth factors for at least 7 days prior to study entry: ANC ≥ 0.75 × 109/L and platelet count ≥ 20 × 109/L.
  • Willingness to avoid pregnancy or fathering children.

Who cannot join

  • Received more than 1 prior allo-SCT. Prior autologous HCT is allowed.
  • Has overlap cGVHD, defined as simultaneous presence of features or characteristics of aGVHD in a patient with cGVHD.
  • Received previous systemic treatment for cGVHD, including systemic corticosteroids and extracorporeal photopheresis.
  • Received systemic corticosteroids within 2 weeks prior to C1D1, regardless of indication.
  • Initiated systemic treatment with CNIs or mTOR inhibitors within 2 weeks prior to C1D1.
  • Prior treatment with a JAK inhibitor within 8 weeks before randomization. Participants who received a JAK inhibitor for the treatment of aGVHD are eligible only if they achieved a response (CR or PR) to JAK inhibitor treatment and did not discontinue due to toxicity.
Show all 15Show fewer
  • Evidence of relapse of the primary hematologic disease or treatment for relapse after the allo-SCT was performed, including DLIs for the treatment of molecular relapse.
  • History of acute or chronic pancreatitis.
  • History of thromboembolic events (such as deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction) in the 6 months prior to study entry.
  • Active symptomatic myositis.
  • Severe renal impairment, that is, estimated CrCl < 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. Participants with CrCl of 30 to 59 mL/min on treatment with fluconazole are not eligible.
  • 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.
  • Currently active significant cardiac disease, such as uncontrolled arrhythmias, uncontrolled hypertension, or Class 3 or 4 congestive heart failure as defined by New York Heart Association, or a history of myocardial infarction or unstable angina within 6 months prior to randomization.
  • Pregnant or breastfeeding.
  • Other protocol-defined Inclusion/Exclusion Criteria may apply.

Trial locations and contacts

68 sites in 7 countries.

68 sites in 7 countries

United States25 sites

  • University of Colorado Cancer Center

    Aurora, Colorado, 80045

  • University of Maryland-Greenebaum Cancer Center

    Baltimore, Maryland, 21201

  • Northwestern University

    Chicago, Illinois, 60611

  • The University of Chicago Medicine

    Chicago, Illinois, 60637

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • The Ohio State University

    Columbus, Ohio, 43210

  • Colorado Blood Cancer Institute

    Denver, Colorado, 80218

  • City of Hope Medical Center

    Duarte, California, 91010

  • Md Anderson Cancer Center

    Houston, Texas, 77030

  • Mayo Clinic Jacksonville

    Jacksonville, Florida, 32224

  • University of California-Los Angeles Medl Cntr-Oncology Center Bowyer Clinic

    Los Angeles, California, 90095

  • Froedtert & the Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Smilow Cancer Center-Yale

    New Haven, Connecticut, 06510

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10021

  • Weill Cornell Medicine

    New York, New York, 10021

  • Mount Sinai Hospital

    New York, New York, 10029

  • Fred and Pamela Buffett Cancer Center

    Omaha, Nebraska, 68198

  • University of Pennsylvania Abramson Cancer Center

    Philadelphia, Pennsylvania, 19104

  • Mayo Clinic Hospital

    Phoenix, Arizona, 85054

  • Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109

  • University of Washington

    Seattle, Washington, 98195

  • Washington University

    St Louis, Missouri, 63110

  • Stanford Cancer Center

    Stanford, California, 94305

Other trials in United States

Spain13 sites

  • Hospital Clinic Barcelona Main

    Barcelona, 08036

  • Hospital Universitario Virgen de La Arrixaca

    El Palmar, 30120

  • Hospital Universitario Virgen de Las Nieves

    Granada, 18014

  • Ico Institut Catala D Oncologia

    Granvia de L'hospitalet 199-203, 08908

  • Hospital General Universitario Gregorio Maranon

    Madrid, 28007

  • Hospital Universitario Ramon Y Cajal

    Madrid, 28034

  • Hospital Universitario 12 de Octubre

    Madrid, 28041

  • Hospital Regional Universitario de Malaga

    Málaga, 29011

  • Hospital Clinico Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario Marques de Valdecilla

    Santander, 39008

  • Hospital Universitario Virgen Del Rocio

    Seville, 41013

  • Hospital Clinico Universitario de Valencia

    Valencia, 46010

  • Hospital Universitari I Politecnic La Fe

    Valencia, 46026

Other trials in Spain

Belgium7 sites

  • Az Sint-Jan Brugge - Oostende Av - Campus Sint-Jan

    Bruges, 08000

  • Universitair Ziekenhuis Antwerpen (Uza)

    Edegem, 02650

  • Jessa Ziekenhuis

    Hasselt, 03500

  • Universitair Ziekenhuis (Uz) Leuven

    Leuven, 03000

  • Universitaire Ziekenhuis Leuven - Gasthuisberg

    Leuven, 03000

  • Centre Hospitalier Universitaire (Chu) de Liege

    Liège, 04000

  • AZ DELTA

    Roeselare, 08800

Other trials in Belgium

Italy7 sites

  • Azienda Socio Sanitaria Territoriale Papa Giovanni Xxiii

    Bergamo, 24127

  • Aou Policlinico S. Orsola-Malpighi

    Bologna, 40138

  • Fondazione Irccs Ca Granda Ospedale Maggiore Policlinico Di Milano Uo Dermatologia

    Milan, 20122

  • Ospedale Pediatrico Bambino Gesu Irccs

    Rome, 00165

  • Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore

    Rome, 00168

  • Irccs Istituto Clinico Humanitas

    Rozzano, 20089

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

    Torino, 10126

Other trials in Italy

Germany6 sites

  • Klinikum Der Johann Wolfgang Goethe University

    Frankfurt am Main, 60590

  • Universitatklinikum Freiburg

    Freiburg I. Breisgau, 79106

  • Universitatsklinikum Hamburg Eppendorf

    Hamburg, 20251

  • Universitaetsklinikum Jena

    Jena, 07747

  • University Hospital Mannheim

    Mannheim, 68167

  • Universitaetsklinikum Regensburg

    Regensburg, 93053

Other trials in Germany

United Kingdom6 sites

  • Queen Elizabeth Hospital

    Birmingham, B15 2TH

  • Cambridge University Hospitals Nhs Foundation Trust

    Cambridge, CB2 0QQ

  • University Hospital of Wales

    Cardiff, CF14 4XW

  • Queen Elizabeth University Hospital

    Glasgow, G51 4TF

  • St James University Hospital

    Leeds, LS9 7TF

  • Clatterbridge Cancer Center

    Liverpool, L7 8YA

Other trials in United Kingdom

Canada4 sites

  • Arthur J E Child Comprehensive Cancer Centre

    Calgary, Alberta, T3N 4N1

  • Chu Sainte-Justine

    Montreal, Quebec, H3T 1C5

  • Princess Margaret Cancer Centre - University Health Network

    Toronto, Ontario, M5G 2M9

  • Vancouver General Hospital

    Vancouver, V5Z1M9

Other trials in Canada

About this study

Official title: A Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease

This study will be conducted to determine the preliminary efficacy of axatilimab in combination with ruxolitinib and to assess the contribution of axatilimab to the combination treatment effect in participants with cGVHD.

Being tested
Axatilimab, Ruxolitinib, Corticosteroids
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.

  • AxatilimabDrug

    Axatilimab will be administered at protocol defined dose.

  • RuxolitinibDrug

    Ruxolitinib will be administered at protocol defined dose.

  • CorticosteroidsDrug

    Also called prednisone, prednisolone, methylprednisolone

    Corticosteroids will be administered at protocol defined dose.

Study groups in NCT06388564
Study groupRoleWhat they receive
Treatment Group AExperimentalAxatilimab will be administered at a protocol defined starting dose plus ruxolitinib at a protocol defined starting dose.
Receives: Drug: Axatilimab; Drug: Ruxolitinib
Treatment Group BExperimentalRuxolitinib will be administered at a protocol defined starting dose.
Receives: Drug: Ruxolitinib
Treatment Group CExperimentalCorticosteroids alone will be administered at a protocol defined starting dose.
Receives: Drug: Corticosteroids

What is being measured

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

  1. Objective Response Rate

    Defined as Complete Response (CR) or Partial Response (PR) at 6 months in the absence of new systemic therapy for cGVHD. Response assessment will be based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

    Measured: 6 months

Secondary outcomes (10)

  1. 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 2 years and 30 days

  2. Duration of Response

    Defined as the time from the first response (PR or CR) to the date of progression of cGVHD, start of new systemic therapy or death from any cause.

    Measured: Up to 2 years

  3. Proportion of participants with a ≥ 7-point improvement in modified Lee symptom scale (mLSS) score

    Measured: Up to 2 years

  4. Best overall response in the first 6 months

    Define as PR or CR in the first 6 months.

    Measured: Up to 6 months

  5. OR at 12 months, defined as CR or PR at 12 months (C14D1) in the absence of new systemic therapy for cGVHD.

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

    Measured: 12 months

  6. Proportion of participants who remain corticosteroid-free

    Measured: 4 weeks, 8 weeks and 6 months

  7. Organ-specific response in the first 6 cycles and on study, based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

    Based on the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.

    Measured: Up to 2 years

  8. Failure-free Survival (FFS)

    Defined as the time from date of randomization to date of initiation of a new cGVHD treatment, malignancy relapse, or death due to any cause.

    Measured: Up to 2 years and 30 days

  9. Axatilimab pharmacokinetic (PK) in Plasma

    Axatilimab concentration in plasma.

    Measured: Up to 2 years and 30 days

  10. Ruxolitinib PK in Plasma

    Ruxolitinib concentration in plasma.

    Measured: Up to 2 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 posted29 Apr 2024
  2. Study start11 Oct 2024
  3. Primary completion1 Oct 2026
  4. Study completion30 Mar 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
130 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06388564
Sponsor's protocol ID
INCA34176-254
Other IDs
2022-502168-19-00
Status last verified
Oct 2026
First submitted
25 Apr 2024
Last updated
15 Oct 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
Expanded access
Available

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Axatilimab, Ruxolitinib and Corticosteroids in people with Chronic Graft-versus-host-disease, run by Incyte Corporation at 68 sites in 7 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 5 inclusion and 15 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 15 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 68 sites in United States, Spain, Belgium, Italy, Germany and 2 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 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?

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 Rate (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 NCT06388564.

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 15 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.

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