Actively Recruiting
A Phase 1b/2 Open-Label Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory Active Chronic Graft-Versus-Host Disease After Systemic Therapy
Led by Incyte Corporation · Updated on 2026-02-03
30
Participants Needed
9
Research Sites
78 weeks
Total Duration
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AI-Summary
What this Trial Is About
Researchers are evaluating axatilimab monotherapy in Chinese participants aged 12 years and older who have recurrent or refractory active chronic graft-versus-host disease (cGVHD) after receiving systemic therapy. The study aims to assess the safety, effectiveness, and pharmacokinetics of this treatment in managing cGVHD, a condition that can occur after stem cell transplantation. This is a Phase 1b/2, open-label, multicenter study sponsored by Incyte Corporation. Participants will receive axatilimab through intravenous infusion at protocol-defined doses. The study includes two parts: Part 1 focuses on safety evaluation, and Part 2 assesses efficacy. The treatment duration and dosing schedule follow the study protocol, and participants may continue to receive corticosteroids, calcineurin inhibitors, or mTOR inhibitors if stable. The study will monitor participants for up to two years and 30 days to gather information on treatment responses and adverse events. Throughout the study, participants will undergo various assessments including monitoring for treatment-emergent adverse events, evaluation of response rates, symptom improvement using the modified Lee symptom scale, and pharmacokinetic sampling for axatilimab. Additional laboratory tests will measure changes in immune markers and antibody development. Participants are expected to attend regular visits for these evaluations and safety monitoring during the study period, which may last up to two years and beyond.
CONDITIONS
Brief Title
A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- At least 12 years old at the time of signing informed consent.
- Ability and willingness to sign the study consent form; parental consent and participant assent required for ages 12 to 17.
- Chinese participants who have undergone allogeneic stem cell transplant and have active, refractory, or recurrent chronic graft-versus-host disease requiring systemic immune suppression despite prior therapy.
- Active cGVHD defined by 2014 NIH criteria.
- Refractory disease showing new or progressing symptoms despite treatment or no response within 3 months.
- Recurrent cGVHD with symptomatic disease requiring new therapy.
- May have overlapping active acute and chronic GVHD symptoms.
- Karnofsky or Lansky performance score of 60 or higher.
- Adequate organ and bone marrow function within 14 days before treatment.
- Stable dose of corticosteroids, calcineurin inhibitors, or mTOR inhibitors for at least 2 weeks if used.
- Willingness to avoid pregnancy or fathering children during and shortly after the study period with appropriate precautions.
You will not qualify if you...
- Presence of acute GVHD without chronic GVHD symptoms.
- Evidence of relapse of underlying cancer or post-transplant lymphoproliferative disease at screening.
- History of acute or chronic pancreatitis.
- Active symptomatic myositis.
- Severe illness, uncontrolled infection, allergy to study drug components, or other conditions making participation unsuitable.
- Positive HIV status.
- History of latent or active tuberculosis or recent close contact with active TB.
- Positive TB tests at screening.
- Active hepatitis B or C infection requiring treatment or risk of reactivation.
- Diagnosis of another malignancy within 3 years unless treated with curative intent and approved.
- Pregnant or breastfeeding.
- Previous exposure to CSF-1R targeted therapies.
- Use of other agents for cGVHD treatment aside from corticosteroids, CNIs, or mTOR inhibitors within 2 weeks or 5 half-lives prior to treatment.
- Receipt of investigational treatment within 28 days before starting study treatment.
- Current participation in another interventional study.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 7 cycles (approximately 169 days)
Participants receive axatilimab monotherapy through intravenous infusion at the protocol-defined dose to treat chronic graft-versus-host disease.
Multiple visits for infusions and assessments during treatment cycles
Duration - Up to 2 years
Participants are monitored for safety and response after completing treatment, including assessment of long-term outcomes up to 2 years.
Periodic visits for follow-up assessments
Trial Site Locations
Total: 9 locations
1
Peking University People'S Hospital
Beijing, China, 101149
Actively Recruiting
2
West China Hospital of Sichuan University
Chengdu, China, 610041
Actively Recruiting
3
The Second Affiliated Hospital, Army Medical University (Xinqiao Hospital)
Chongoing, China, 400037
Actively Recruiting
4
Zhujiang Hospital of Southern Medical University
Guangzhou, China, 510280
Actively Recruiting
5
Nanfang Hospital of Southern Medical University
Guangzhou, China, 510515
Actively Recruiting
6
The First Affiliated Hospital, Zhejiang University School of Medicine
Hangzhou, China, 310024
Actively Recruiting
7
Shanghai Children'S Medical Center
Shanghai, China, 200127
Actively Recruiting
8
Institute of Hematology, Chinese Academy of Medical Sciences
Tianjin, China, 301617
Actively Recruiting
9
Union Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology
Wuhan, China, 430022
Actively Recruiting
Research Team
I
Incyte Corporation Call Center (US)
I
Incyte Corporation Call Center (ex-US)
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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