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Monoclonal Antibodies

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

Phase 2

Waitlist Available

Research Sponsored by Syndax Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Karnofsky Performance Scale of ≥60 (if aged 16 years or older); Lansky Performance Score of ≥60 (if aged <16 years)

Participants who are allogeneic hematopoietic stem cell transplantation (HSCT) recipients with active cGVHD requiring systemic immune suppression

Must not have

Taking agents for treatment of cGVHD other than corticosteroids or either a CNI or mTOR inhibitor is prohibited

Previous exposure to CSF1-R targeted therapies

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug, axatilimab, to see if it is effective, safe, and tolerated in people with cGVHD who have had two or more prior lines of systemic therapy.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions related to axatilimab across the different dosages being tested.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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

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My kidney function, measured by creatinine clearance, is adequate.

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My chronic GVHD hasn't improved after two different treatments.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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

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I have been treated with CSF1-R targeted therapies before.

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I am currently pregnant or breastfeeding.

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I have acute graft-versus-host disease without signs of chronic GVHD.

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I have had pancreatitis before.

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I have a history of muscle inflammation.

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My cancer has returned or worsened after treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Overall Response Rate (ORR) in the First 6 Cycles as Defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-Versus-Host Disease (cGVHD)

Secondary study objectives

Duration of Response

Organ-specific Response Rate

Sustained Response Rate