Axatilimab for Graft-versus-Host Disease
(AGAVE-201 Trial)
Trial Summary
What is the purpose of this trial?
This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received at least 2 prior lines of systemic therapy.
Will I have to stop taking my current medications?
You may need to stop taking some of your current medications. If you're on treatments for cGVHD other than corticosteroids, CNI, or mTOR inhibitors, you'll need a washout period (time without taking certain medications) of 2 weeks or 5 half-lives, whichever is shorter, before joining the study.
What data supports the effectiveness of the drug Axatilimab for treating graft-versus-host disease?
Is Axatilimab safe for humans?
Axatilimab has been studied in a Phase I/II trial for chronic graft-versus-host disease, and it is a humanized monoclonal antibody that targets specific immune cells. While the study focused on its effectiveness, the trial's completion suggests it was considered safe enough to proceed through these phases.56789
What makes the drug Axatilimab unique for treating graft-versus-host disease?
Axatilimab is unique because it is a humanized monoclonal antibody that targets and inhibits the colony-stimulating factor 1 receptor (CSF-1R), which helps reduce the development of macrophages that contribute to chronic graft-versus-host disease (cGVHD) fibrosis. This mechanism of action is different from other treatments that focus on T-cell modulation.57101112
Research Team
Vedran Radojcic, M.D.
Principal Investigator
Syndax Pharmaceuticals
Eligibility Criteria
This trial is for people aged 2 or older with chronic graft versus host disease (cGVHD) who've had at least two prior systemic therapies. They must have a certain level of physical function, adequate organ and bone marrow function, and not be pregnant or breastfeeding. Participants can't join if they're on other cGVHD treatments besides corticosteroids/CNI/mTOR inhibitors, in another study, have been exposed to CSF1-R targeted therapies, or have severe illnesses.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive axatilimab at 3 different doses in 28-day treatment cycles
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Axatilimab
Axatilimab is already approved in United States for the following indications:
- Chronic Graft-Versus-Host Disease
Find a Clinic Near You
Who Is Running the Clinical Trial?
Syndax Pharmaceuticals
Lead Sponsor