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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04710576 | A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD) | PHASE2 | ACTIVE NOT_RECRUITING | 241 | — | — | Mar 4, 2021 | Sep 1, 2027 | Mar 16, 2026 | 121 | United States, Australia +14 |
| NCT03604692 | A Phase 1/2 Study to Evaluate Axatilimab in Participants With Active cGVHD | PHASE1 | COMPLETED | 41 | — | — | Nov 1, 2018 | Oct 18, 2024 | Sep 15, 2025 | 11 | United States |
The ORR was defined as the percentage of participants with objective response (complete response \[CR\] or partial response \[PR\]). CR was defined as resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site.
A DLT was defined as the occurrence of any protocol-specified event within the first 28 days from the first dose of SNDX-6352 or administration of the third dose (Cycle 2 Day 1), whichever is later (from Cycle 1 Day 1 to Cycle 2 Day 1) and assessed by the Investigator as not being definitely attributable to underlying disease, disease progression, inter-current illness, concomitant medications or any other alternative cause.
The RP2D was determined in discussion with the Sponsor, Medical Monitor, and Dose Determination Phase Investigators and was based on observations from the Phase 1 of the study (clinical benefit in chronic graft versus host disease \[cGVHD\] and pharmacokinetic/pharmacodynamic effects).
CR or PR was defined by the 2014 National Institutes of Health (NIH) Consensus Development Project on Criteria for Clinical Trials in cGVHD.CR was defined as resolution of all manifestations in each organ or site, and PR was defined as improvement in at least 1 organ or site without progression in any other organ or site. ORR was defined as the percentage of participants achieving a best overall response of CR or PR .ORR calculated as: (number of participants with best overall response as CR or PR)/total number of participants.
| Arm | Type | Description |
|---|---|---|
| Axatilimab Dose Cohort 1 | EXPERIMENTAL | Participants will be administered axatilimab 0.3 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks for up to 2 years. |
| Axatilimab Dose Cohort 2 | EXPERIMENTAL | Participants will be administered axatilimab 1 mg/kg IV every 2 weeks for up to 2 years. |
| Axatilimab Dose Cohort 3 | EXPERIMENTAL | Participants will be administered axatilimab 3 mg/kg IV every 4 weeks for up to 2 years. |
| Cohorts of escalating dose levels of axatilimab | EXPERIMENTAL | Escalating dose levels of axatilimab to establish the optimal biologic dose (OBD) and recommended Phase 2 dose (RP2D). Intravenous (IV) infusion; axatilimab at a dose of 0.15 milligrams (mg)/kilogram (kg) to 3 mg/kg. |
| Phase 2 Dose Expansion | EXPERIMENTAL | Phase 2, dose expansion, is an open-label design, evaluating the 1 mg/kg dose in a larger sample size. IV infusion; axatilimab at a dose of 1 mg/kg. |
| Name | Type | Description |
|---|---|---|
| Axatilimab | DRUG | Axatilimab is a high-affinity antibody targeting the colony stimulating factor 1 receptor (CSF-1R). CSF-1R signaling has been demonstrated in nonclinical studies to be the key regulatory pathway involved in the expansion and infiltration of donor-derived macrophages that mediate the disease processes involved in cGVHD. |
Inclusion Criteria: 1. Participants must be 2 years of age or older, at the time of signing the informed consent. 2. Participants who are allogeneic hematopoietic stem cell transplantation (HSCT) recipients with active cGVHD requiring systemic immune suppression. Active cGVHD is defined as the pres...