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Chronic Graft Versus Host DiseasePhase 2NCT06663722

Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease

A Phase 2 study testing Axatilimab and Extracorporeal Photopheresis in people with Chronic Graft Versus Host Disease and cGVHD, run by University of Miami at 3 sites in United States.

Recruiting

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

Sites and contacts
Official record
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Trial summary

The purpose of this study is to see whether giving participants a combination treatment of Axatilimab and Extracorporeal Photopheresis (ECP) is effective against chronic Graft-versus-Host Disease (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, cGVHD
What is being tested?
Axatilimab and Extracorporeal Photopheresis
Could I receive a placebo?
No, everyone receives an active treatment
How many people
49 participants
Where
3 sitesUnited States
Study dates
5 May 2025to 5 May 2030
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

  • Recipient of allogeneic hematopoietic cell transplantation (HCT).
  • Age greater or equal to 12.
  • Chronic GVHD per 2014 National Institutes of Health Consensus Criteria (NCC) (Jagasia et al. 2015) or overlap syndrome requiring new therapy in patients with at least 2 prior lines of therapy, steroid refractoriness, or steroid dependence:
  • Prior systemic lines of therapy may include corticosteroids, calcineurin inhibitor (CNI) or sirolimus, or other systemic immunosuppressive agent such as ruxolitinib, belumosudil, or ibrutinib. GVHD prophylaxis does not count as a prior line of therapy.
  • Steroid refractory is defined as any of the following criteria:
  • i. Manifestations progress despite the use of ≥ 1 mg/kg/day prednisone for at least 1 week
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  • ii. Manifestations persist without improvement despite treatment with ≥ 0.5 mg/kg/day or 1 mg/kg every other day for at least four weeks.
  • iii. Recurrence after a CR, or
  • iv. Progression after a PR.
  • Steroid dependence is defined as inability to control cGVHD symptoms while tapering prednisone below 0.25 mg/kg/day on at least two occasions separated by at least 8 weeks. There must be evidence of clinically active cGVHD.
  • For patients receiving approved or commonly used agents, all GVHD systemic treatments should be discontinued except for corticosteroids and drugs being continued from GVHD prophylaxis at screening.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-3 as assessed at Screening.
  • Platelet count > 50,000 platelets/μL and absolute neutrophil count > 1,000 cells/μL as measured at Screening.
  • Aspartate aminotransferase (AST), alanine aminotransferase (ALT) ≤ 2.5 x upper limit of normal (ULN), unless attributed to presumed cGVHD as measured at Screening.
  • Stable dose of corticosteroids for at least 14 days prior to treatment.
  • Sexually mature individuals must use contraception as described in Section 4.12. For individuals less than 18 years of age, sexual maturity will be determined as per treating pediatrician.

Who cannot join

  • Pregnancy or breast-feeding.
  • Active relapse of underlying malignancy.
  • History or the presence of interstitial pneumonitis or drug-related pneumonitis.
  • Active gastrointestinal (GI) bleeding.
  • Inability to tolerate volume shifts associated with ECP (e.g., inadequate renal, hepatic, pulmonary and cardiac function (ejection fraction (EF) < 40%) per Investigator discretion.
  • History of myositis.
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  • History of splenectomy.
  • History of pancreatitis.
  • History of other malignancy (within 3 years of Screening) unless treated with curative intent and approved by Principal Investigator (PI).
  • Significant, uncontrolled, or active comorbid conditions or are unable to adhere to the study requirements.
  • Acquired Immune Deficiency Syndrome (AIDS) or active hepatitis B (Hep B) or active hepatitis C (Hep C) infection.
  • Prior colony-stimulating factor-1 (CSF-1R) targeted therapies.
  • Prior history of ECP treatment failure or intolerance.
  • Intolerance to methoxsalen, heparin, or citrate products.
  • Patients with aphakia due to risk of increased retinal damage or photosensitive disease (albinism, systemic lupus erythematosus, porphyria).
  • Lack of stable IV access. Acceptable forms include central venous catheter, peripherally inserted central catheter (PICC), or peripheral IV line per institutional guidelines.
  • Insurance denial of coverage for the ECP procedure.

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Trent P Wang, DO contact

+1 (305) 2436444

trentwang@med.miami.edu

United States3 sites, 3 recruiting

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

    Jennifer Schneiderman, MD, MS, jschneiderman@luriechildrens.org

    Jennifer Schneiderman, MD,MS

    Recruiting

  • University of Miami

    Miami, Florida, 33136

    Trent P Wang, DO, 305-243-6444, trentwang@med.miami.edu

    Trent P Weng, DO

    Recruiting

  • University of California, San Francisco

    San Francisco, California, 94143

    Jerry Lee, MD, MS, jerry.lee2@ucsf.edu

    Jerry Lee, MD, MS

    Recruiting

Other trials in United States

About this study

Official title: A Phase II b Study of Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease

The purpose of this study is to see whether giving participants a combination treatment of Axatilimab and Extracorporeal Photopheresis (ECP) is effective against chronic Graft-versus-Host Disease (cGVHD).

Being tested
Axatilimab, Extracorporeal Photopheresis
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.

  • AxatilimabBiological

    Axatilimab will be administered intravenously (IV) at a dose of 0.3 mg/kg, beginning as a pre-phase dose two weeks prior to initiation of Extracorporeal Photopheresis (ECP) therapy. Thereafter, Axatilimab will be administered with a frequency of one treatment session bi-weekly during each treatment cycle.

  • Extracorporeal PhotopheresisProcedure or surgery

    Also called ECP

    Mandatory ECP therapy will be administered at a frequency of two treatment sessions per week during Cycles 1 through 3, two treatment bi-weekly during Cycles 4 through 6, and two treatments during week 1 of Cycle 7.

    Optional ECP therapy will be administered at a frequency of two treatment sessions during weeks 2 and 4 of Cycles 4 through 6, when mandatory ECP is not administered. Optional ECP therapy will also be administered as two treatment sessions during week 3 of Cycle 7.

    After Cycle 7, participants may receive ECP therapy only at the Investigator's discretion for a maximum Treatment Period of 12 months.

Study groups in NCT06663722
Study groupRoleWhat they receive
Axatilimab in combination with ECP GroupExperimentalParticipants in this group will receive Axatilimab in combination with extracorporeal photopheresis (ECP) therapy for up to seven (7) four-week cycles. Total participation duration is about 15 months.
Receives: Biological: Axatilimab; Procedure: Extracorporeal Photopheresis

What is being measured

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

  1. Best Overall Response Rate (ORR)

    Best overall response rate (ORR) will be reported as the percentage of participants who achieve partial response (PR) or a complete response (CR) to study therapy, as defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in chronic graft-versus-host disease (cGVHD) while on study treatment.

    Measured: Up to 24 weeks

Secondary outcomes (8)

  1. Proportion of participants experiencing treatment-related adverse events (AEs)

    Proportion of participants experiencing treatment-related adverse events (AEs) will be reported. AEs, including serious adverse events (SAEs), will be assessed using the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0. Attribution of AEs and SAEs to study treatment will be reported as possibly, probably and definitely related, as determined by the treating physician.

    Measured: Up to 15 months

  2. Proportion of participants experiencing serious adverse events (SAEs)

    Proportion of participants experiencing serious adverse events (SAEs) will be reported, regardless of attribution to study treatment, as determined by the treating physician. SAEs will be assessed using the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.

    Measured: Up to 15 months

  3. Change in cumulative dose of corticosteroid usage

    Change in cumulate dose corticosteroid usage among study participants will be reported, from baseline to 24 weeks and to 1-year post start of Axatilimab. Corticosteroid dosage, recorded as average dose in mg per day, will be recorded at each interval.

    Measured: Baseline, 24 weeks, 1 year

  4. Duration of response (DOR)

    Duration of response (DOR) is defined as the elapsed time in months from best response (partial response (PR) or complete response (CR)) to documented progression of chronic graft-versus-host disease (cGVHD), start of new therapy, or death for any reason.

    Measured: Up to 15 months

  5. Relapse-free survival (RFS)

    Relapse-free survival (RFS) is defined as the elapsed time in months from the date of the first dose of study treatment to relapse or recurrence of the primary hematologic malignancy or disorder or death.

    Measured: Up to 15 months

  6. Change in Quality of life (QoL) as measured by the modified Lee Symptom Scale (mLSS) score

    Changes in quality of life (QoL) from baseline, as measured by the modified Lee Symptom Scale (mLSS) score questionnaire, will be estimated at 24 weeks and 1 year and reported. The mLSS is a 30-item self-reported questionnaire, with seven (7) subscales (skin, eyes, mouth, lung, nutrition, energy and psych) containing 2-7 items which allow calculation of a summary score. Each item uses a 5-point Likert scale ranging from 0 points ("Not at all"/"No symptoms") to 4 points ("Extremely"/"Very severe"). Lower scores indicate improved quality of life. An improvement in > 5-points is considered clinically significant.

    Measured: Baseline, 24 weeks, 1 year

  7. Proportion of participants who develop subsequent sclerotic skin disease

    The proportion of participants who develop subsequent sclerotic skin disease in those who present without such manifestations will be reported.

    Measured: Up to 15 months

  8. Rate of Complete Response (CR) at Best Response

    The rate of complete response (CR) at best response will be reported as the percentage of patients who achieve CR at best response, as defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in chronic graft-versus-host disease (cGVHD) while on study treatment.

    Measured: Up to 24 weeks

Timeline

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

  1. First posted29 Oct 2024
  2. Study start5 May 2025
  3. Primary completion5 May 2030 (estimated)
  4. Study completion5 May 2030 (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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
49 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06663722
Sponsor's protocol ID
20240116
Status last verified
Jun 2026
First submitted
26 Oct 2024
Last updated
22 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Axatilimab and Extracorporeal Photopheresis in people with Chronic Graft Versus Host Disease and cGVHD, run by University of Miami at 3 sites in United States.

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 16 inclusion and 17 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 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. 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 Trent P Wang, DO (+1 (305) 2436444, trentwang@med.miami.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Best Overall Response Rate (ORR) (time frame: Up to 24 weeks). 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 NCT06663722.

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 22 Jun 2026.

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