Axatilimab for Graft-versus-Host Disease
(AGAVE-201 Trial)

Recruiting in Palo Alto (17 mi)

+124 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Syndax Pharmaceuticals

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

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.

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

I can care for myself but may not be able to do active work or play.

My organs and bone marrow are functioning well.

I had a stem cell transplant and need treatment for ongoing graft-versus-host disease.

+8 more

Exclusion Criteria

I haven't had any cancer other than the one I'm seeking treatment for in the last 3 years, or if I have, it was treated with the intent to cure and is approved by the study's medical team.

I am not taking any cGVHD treatments other than steroids, CNI, or mTOR inhibitors.

Receiving another investigational treatment within 28 days of randomization

+10 more

Participant Groups

The study tests the effectiveness and safety of axatilimab at three different doses in patients with active cGVHD that's come back or hasn't responded to treatment after an allogeneic stem cell transplant. It's a Phase 2 trial focusing on those who've tried multiple treatments already.

3Treatment groups

Experimental Treatment

Group I: Axatilimab Dose Cohort 3Experimental Treatment1 Intervention

Participants will be administered axatilimab 3 mg/kg IV every 4 weeks for up to 2 years.

Group II: Axatilimab Dose Cohort 2Experimental Treatment1 Intervention

Participants will be administered axatilimab 1 mg/kg IV every 2 weeks for up to 2 years.

Group III: Axatilimab Dose Cohort 1Experimental Treatment1 Intervention

Participants will be administered axatilimab 0.3 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks for up to 2 years.

Axatilimab is already approved in United States for the following indications:

🇺🇸 Approved in United States as Axatilimab for: