Authorised Therapeutic confirmatory (Phase III) Chronic Graft-Versus-Host Disease

A Phase 3, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy

EU CTIS ID: 2024-518973-32-00

What this study is testing

To compare the efficacy of axatilimab monotherapy versus BAT in cGVHD

Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

1. Age ≥ 12 years at the time of signing the ICF.
2. Ability to comprehend and willingness to sign a written ICF for the study. A parent/guardian should provide consent for pediatric participants unable to provide consent themselves; in addition, where applicable, pediatric participants should sign their own assent form.
10. Willingness to avoid pregnancy or fathering children
3. Active, moderate to severe cGVHD requiring systemic immune suppression.
4. History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood).
5. Participants with refractory or recurrent active cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib.

You likely can't join if

1. Receipt of more than 1 prior allo-HCT.
10. Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
11. Previous exposure to CSF-1R–targeted therapies.
12. For approved or commonly used treatments for cGVHD (other than corticosteroids, CNI, and mTOR inhibitor) a washout period of 2 weeks or 5 half-lives, whichever is longer, is required at study enrollment.
13. Treatment with an investigational agent (for any indication) within 30 days of randomization or within 5 half-lives of the investigational product, whichever is longer.
14. Active, uncontrolled infection despite appropriate therapy at the time of screening.

See the full eligibility criteria

Who can join

1. Age ≥ 12 years at the time of signing the ICF.
2. Ability to comprehend and willingness to sign a written ICF for the study. A parent/guardian should provide consent for pediatric participants unable to provide consent themselves; in addition, where applicable, pediatric participants should sign their own assent form.
10. Willingness to avoid pregnancy or fathering children
3. Active, moderate to severe cGVHD requiring systemic immune suppression.
4. History of allo-HCT from any donor HLA type (related or unrelated donor with any degree of HLA matching) using any graft source (bone marrow, peripheral blood stem cells, or cord blood).
5. Participants with refractory or recurrent active cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib.
6. Participants may have overlap cGVHD (presence of features or characteristics of aGVHD with simultaneous diagnostic and/or distinctive features of cGVHD, per NIH 2014 consensus criteria for cGVHD).
7. KPS score of ≥ 60 (if aged 16 years or older); LPS score of ≥ 60 (if aged < 16 years).
8. Concomitant use of systemic corticosteroids is allowed.
9. Participants must accept to be treated with one of the following BAT options on C1D1: CNI (cyclosporine or tacrolimus), ECP, MMF, an mTOR inhibitor (everolimus or sirolimus), rituximab, pentostatin, proteasome inhibitors, imatinib, or ibrutinib.

What rules you out

1. Receipt of more than 1 prior allo-HCT.
10. Systemic treatment with CNIs or mTOR inhibitors started within 2 weeks prior to C1D1.
11. Previous exposure to CSF-1R–targeted therapies.
12. For approved or commonly used treatments for cGVHD (other than corticosteroids, CNI, and mTOR inhibitor) a washout period of 2 weeks or 5 half-lives, whichever is longer, is required at study enrollment.
13. Treatment with an investigational agent (for any indication) within 30 days of randomization or within 5 half-lives of the investigational product, whichever is longer.
14. Active, uncontrolled infection despite appropriate therapy at the time of screening.
15. Active HBV or HCV infection that requires treatment or at risk for HBV reactivation (ie, positive HbsAg). Participants with pretransplant positive total HBc antibody or positive HCV antibody must have negative viral load for HBV and HCV at screening.
16. Known HIV seropositive status.
17. Suspected active or latent tuberculosis (as confirmed by a positive QuantiFERON® test [QIAGEN, Venlo, The Netherlands] or other tuberculosis blood test).
18. Administration of live-attenuated vaccines within 4 weeks prior to the first dose of study treatment or anticipated need for live-attenuated vaccines while on study treatment.
19. Is pregnant or breastfeeding.
2. Has aGVHD without manifestations of cGVHD.
20. Participation in any other interventional study.
21. Any condition that would, in the investigator's judgment, interfere with full participation in the study, including administration of study drug and attending required study visits, pose a significant risk to the participant, or interfere with interpretation of study data.
22. The following participants are excluded in France: vulnerable populations according to article L.1121-6 of the French Public Health Code and adults under legal protection or who are unable to express their consent per article L.1121-8 of the French Public Health Code, not affiliated to a social security per article L.1121-8-1 of the French Public Health Code.
3. Evidence of relapse of hematologic disease or treatment for relapse after the allo-SCT was performed, including DLI for the treatment of molecular relapse.
4. Maintenance therapy for the primary hematologic disease started within 4 weeks before initiation of study treatment (Day 1) or plans to start maintenance therapy after Day 1.
5. Severe renal impairment, that is, estimated creatinine clearance < 30 mL/min measured or calculated by Cockcroft-Gault equation in adults and Schwartz formula in pediatric participants, or end-stage renal disease on dialysis.
6. Impaired liver function, defined as total bilirubin > 1.5 × ULN and/or ALT and AST > 3 × ULN in participants with no evidence of liver cGVHD.
7. History of acute or chronic pancreatitis.
8. Active, symptomatic myositis.
9. Known allergies, hypersensitivity, or intolerance to the study medications, excipients, or similar compounds.

The study team makes the final eligibility decision.

Where it's taking place

Switzerland
United Kingdom

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 0-17 years, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Switzerland; United Kingdom. Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.