Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06821542 | A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy | PHASE3 | ACTIVE NOT_RECRUITING | 9 | — | — | Jun 4, 2025 | Nov 30, 2032 | Mar 23, 2026 | 130 | Austria, Belgium +14 |
| NCT06585774 | A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease | PHASE3 | RECRUITING | 240 | — | — | Jan 21, 2025 | Mar 31, 2030 | Jun 1, 2026 | 122 | United States, Australia +11 |
| NCT06263478 | A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Axatilimab Monotherapy in Japanese Participants With Recurrent or Refractory Active Chronic Graft-Versus-Host Disease | PHASE3 | ACTIVE NOT_RECRUITING | 21 | — | — | Jul 30, 2024 | Mar 30, 2027 | Jan 7, 2026 | 16 | Japan |
| NCT07124078 | A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256) | PHASE2 | RECRUITING | 60 | — | — | May 20, 2026 | Jul 31, 2029 | Jun 2, 2026 | 41 | United States, Belgium +4 |
| NCT06843408 | A Ph1b/2 Study of Axatilimab Monotherapy in Chinese Participants With Recurrent or Refractory cGVHD | PHASE1 | RECRUITING | 30 | — | — | Jun 25, 2025 | Dec 31, 2027 | Feb 3, 2026 | 9 | China |
Defined for each treatment group as complete response (CR) or partial response (PR) at 6 months (Cycle 7 Day 1, 28-day cycles) in the absence of new systemic therapy for cGVHD. Responses defined by the 2014 NIH consensus criteria.
Defined from the date of randomization to the date of any predefined event, whichever occurs first.
The overall response rate will be assessed by the number of participants with objective response by Cycle 7 (28-day cycles), Day 1, with responses defined by the 2014 NIH consensus criteria.
Defined as adverse events reported for the first time or worsening of a pre-existing event, occurring after the first dose.
The overall response rate will be assessed by the number of participants with objective response by Cycle 7 (28-day cycles), Day 1, with responses defined by the 2014 NIH Consensus Development Project on Criteria for Clinical Trials in cGVHD.
| Arm | Type | Description |
|---|---|---|
| Axatilimab | EXPERIMENTAL | Axatilimab at the protocol-defined dose. |
| Best available Treatment (BAT) | EXPERIMENTAL | Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. |
| Axatilimab + Corticosteroids | EXPERIMENTAL | Axatilimab and Corticosteroids at the protocol-defined dose. |
| Placebo + Corticosteroids | EXPERIMENTAL | Matching placebo and Corticosteroids at the protocol-defined dose. |
| Axatilimab Dose | EXPERIMENTAL | Axatilimab at the protocol-defined dose. |
| Part 1 Safety Evaluation | EXPERIMENTAL | Axatilimab at the protocol-defined dose. |
| Part 2 Efficacy Evaluation | EXPERIMENTAL | Axatilimab at the protocol-defined dose. |
| Name | Type | Description |
|---|---|---|
| INCA034176 | DRUG | IV infusion |
| Best Available Therapy (BAT) | DRUG | Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. |
| Placebo | DRUG | IV infusion |
| Corticosteroids | DRUG | Oral/IV Infusion |
| Best available Treatment (BAT) | DRUG | Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. |
Inclusion Criteria: * Age ≥ 12 years at the time of signing the ICF. * Active, moderate to severe cGVHD, requiring systemic immune suppression. * Participants with refractory or recurrent cGVHD who have received at least 2 lines of systemic therapy, including corticosteroids and ruxolitinib. * Conc...