Ongoing clinical trials with FXI and FXII inhibitors
| Agent . | Registry number (name) . | Clinical trial phase and indication . | No. of patients . | Comparator . | Additional therapies . | Efficacy outcome . | Safety outcomes . | Study recruitment status* . |
|---|---|---|---|---|---|---|---|---|
| IONIS FXI-Rx (ISIS 416858) | NCT03358030 (EMERALD) | Phase 2 Patients with ESRD on hemodialysis | 315 | Placebo | NA | PK, PD | Major bleeding or CRNMB, treatment- emergent AEs | Completed, awaiting results |
| Fesomersen (IONIS-FXI-LRx and BAY2976217) | NCT03582462 | Phase 1 Healthy volunteers | 66 | Placebo | NA | PK, PD | Treatment- emergent AEs | Completed, awaiting results |
| NCT04534114 (RE-THINc ESRD) | Phase 2 Prevention of cardiovascular events in patients with ESRD | 307 | Placebo | NA | PK, PD | Major bleeding or CRNMB, treatment- emergent AEs | Active, not recruiting | |
| Antibodies | ||||||||
| Abelacimab (MAA868) | NCT04213807 | Phase 2 Atrial fibrillation | 28 | Placebo | NA | Dose-range finding study—PK, PD | Safety, tolerability, and immunogenicity | Completed, awaiting results |
| NCT04755283 (AZALEA-TIMI 71) | Phase 2 Atrial fibrillation | Estimated enrollment 1200 | Rivaroxaban 15 mg and 20 mg OD | NA | NA | Safety and tolerability, major bleeding, and CRNMB | Active, not recruiting | |
| NCT05171049 (ASTER) | Phase 3 Treatment of cancer- associated VTE | Estimated enrollment 1655 | Apixaban 10 mg BID followed by 5 mg BID | NA | Centrally adjudicated VTE recurrence | Major bleeding or CRNMB | Active, recruiting | |
| NCT05171075 (MAGNOLIA) | Phase 3 Treatment of GI and GU cancer-associated VTE | Estimated enrollment 1020 | Dalteparin 200 IU/kg/d followed by 150 IU/kg/d | NA | Centrally adjudicated VTE recurrence | Major bleeding or CRNMB | Active, recruiting | |
| Osocimab (BAY 1213790) | NCT03787368 | Phase 1 Safety in patients with ESRD | 55 | Placebo | NA | PK, PD | Major bleeding or CRNMB | Completed, awaiting results |
| NCT04523220 (CONVERT) | Phase 2 Safety in patients with ESRD | 686 | Placebo | NA | PK, PD | Major bleeding or CRNMB | Active, not recruiting | |
| Xisomab (AB023) | NCT04465760 | Phase 2 Prevention of CAT in patients with cancer receiving chemotherapy | Estimated enrollment 50 | None | NA | Incidence of CAT | Major bleeding or CRNMB | Active, recruiting |
| Garadacimab (CSL312) | NCT04281524 | Phase 1/2 Prevention of CAT in patients with cancer receiving chemotherapy | 0 (study withdrawn) | Placebo | NA | Incidence of CAT | Treatment- emergent AEs | Withdrawn (business decision, not safety related) |
| NCT04409509 | Phase 2 COVID-19 | 124 | Placebo | NA | Endotracheal intubation or death prior to intubation | Completed, awaiting results | ||
| NCT05130970 | Phase 2 Idiopathic pulmonary fibrosis | Estimated enrollment 80 | Placebo | NA | PK, PD | Treatment-emergent AEs | Active, recruiting | |
| NCT04656418 | Phase 3 HAE-C1-INH | Estimated enrollment 60 | Placebo | NA | Time-normalized number of HAE-C1-INH attacks during treatment | Treatment-emergent AEs | Active, not recruiting | |
| Small molecules | ||||||||
| Milvexian (BMS-986177/JNJ-70033093) | NCT03766581 (AXIOMATIC-SSP) | Phase 2 Secondary stroke prevention | 2366 | Placebo | Aspirin, clopidogrel | Composite of new ischemic stroke during the treatment period and new covert brain infarction on MRI | Bleeding according to BARC criteria | Active, not recruiting |
| Asundexian (BAY 2433334) | NCT04304534 (PACIFIC-AMI) | Phase 2 Acute MI | 1592 | Placebo | Aspirin, clopidogrel | CV events: MI, stroke, stent thrombosis, and death | Bleeding according to BARC criteria | Completed, awaiting results |
| NCT04304508 (PACIFIC-STROKE) | Phase 2 Acute noncardioembolic ischemic stroke | 1808 | Placebo | NA | Symptomatic ischemic stroke or covert brain infarcts on MRI | Major bleeding or CRNMB | Completed, awaiting results | |
| NCT04510987 | Phase 1 Patients with ESRD on hemodialysis | 48 | None | NA | PK, PD | Treatment-emergent AEs | Completed, awaiting results | |
| Agent . | Registry number (name) . | Clinical trial phase and indication . | No. of patients . | Comparator . | Additional therapies . | Efficacy outcome . | Safety outcomes . | Study recruitment status* . |
|---|---|---|---|---|---|---|---|---|
| IONIS FXI-Rx (ISIS 416858) | NCT03358030 (EMERALD) | Phase 2 Patients with ESRD on hemodialysis | 315 | Placebo | NA | PK, PD | Major bleeding or CRNMB, treatment- emergent AEs | Completed, awaiting results |
| Fesomersen (IONIS-FXI-LRx and BAY2976217) | NCT03582462 | Phase 1 Healthy volunteers | 66 | Placebo | NA | PK, PD | Treatment- emergent AEs | Completed, awaiting results |
| NCT04534114 (RE-THINc ESRD) | Phase 2 Prevention of cardiovascular events in patients with ESRD | 307 | Placebo | NA | PK, PD | Major bleeding or CRNMB, treatment- emergent AEs | Active, not recruiting | |
| Antibodies | ||||||||
| Abelacimab (MAA868) | NCT04213807 | Phase 2 Atrial fibrillation | 28 | Placebo | NA | Dose-range finding study—PK, PD | Safety, tolerability, and immunogenicity | Completed, awaiting results |
| NCT04755283 (AZALEA-TIMI 71) | Phase 2 Atrial fibrillation | Estimated enrollment 1200 | Rivaroxaban 15 mg and 20 mg OD | NA | NA | Safety and tolerability, major bleeding, and CRNMB | Active, not recruiting | |
| NCT05171049 (ASTER) | Phase 3 Treatment of cancer- associated VTE | Estimated enrollment 1655 | Apixaban 10 mg BID followed by 5 mg BID | NA | Centrally adjudicated VTE recurrence | Major bleeding or CRNMB | Active, recruiting | |
| NCT05171075 (MAGNOLIA) | Phase 3 Treatment of GI and GU cancer-associated VTE | Estimated enrollment 1020 | Dalteparin 200 IU/kg/d followed by 150 IU/kg/d | NA | Centrally adjudicated VTE recurrence | Major bleeding or CRNMB | Active, recruiting | |
| Osocimab (BAY 1213790) | NCT03787368 | Phase 1 Safety in patients with ESRD | 55 | Placebo | NA | PK, PD | Major bleeding or CRNMB | Completed, awaiting results |
| NCT04523220 (CONVERT) | Phase 2 Safety in patients with ESRD | 686 | Placebo | NA | PK, PD | Major bleeding or CRNMB | Active, not recruiting | |
| Xisomab (AB023) | NCT04465760 | Phase 2 Prevention of CAT in patients with cancer receiving chemotherapy | Estimated enrollment 50 | None | NA | Incidence of CAT | Major bleeding or CRNMB | Active, recruiting |
| Garadacimab (CSL312) | NCT04281524 | Phase 1/2 Prevention of CAT in patients with cancer receiving chemotherapy | 0 (study withdrawn) | Placebo | NA | Incidence of CAT | Treatment- emergent AEs | Withdrawn (business decision, not safety related) |
| NCT04409509 | Phase 2 COVID-19 | 124 | Placebo | NA | Endotracheal intubation or death prior to intubation | Completed, awaiting results | ||
| NCT05130970 | Phase 2 Idiopathic pulmonary fibrosis | Estimated enrollment 80 | Placebo | NA | PK, PD | Treatment-emergent AEs | Active, recruiting | |
| NCT04656418 | Phase 3 HAE-C1-INH | Estimated enrollment 60 | Placebo | NA | Time-normalized number of HAE-C1-INH attacks during treatment | Treatment-emergent AEs | Active, not recruiting | |
| Small molecules | ||||||||
| Milvexian (BMS-986177/JNJ-70033093) | NCT03766581 (AXIOMATIC-SSP) | Phase 2 Secondary stroke prevention | 2366 | Placebo | Aspirin, clopidogrel | Composite of new ischemic stroke during the treatment period and new covert brain infarction on MRI | Bleeding according to BARC criteria | Active, not recruiting |
| Asundexian (BAY 2433334) | NCT04304534 (PACIFIC-AMI) | Phase 2 Acute MI | 1592 | Placebo | Aspirin, clopidogrel | CV events: MI, stroke, stent thrombosis, and death | Bleeding according to BARC criteria | Completed, awaiting results |
| NCT04304508 (PACIFIC-STROKE) | Phase 2 Acute noncardioembolic ischemic stroke | 1808 | Placebo | NA | Symptomatic ischemic stroke or covert brain infarcts on MRI | Major bleeding or CRNMB | Completed, awaiting results | |
| NCT04510987 | Phase 1 Patients with ESRD on hemodialysis | 48 | None | NA | PK, PD | Treatment-emergent AEs | Completed, awaiting results | |
AEs, adverse events; BARC, Bleeding Academic Research Consortium; BID, twice daily; CAT, catheter-associated thrombosis; COVID-19, coronavirus disease 2019; CRNMB, clinically relevant nonmajor bleeding; CV, cardiovascular; HAE-C1-INH, C1-esterase inhibitor-deficient hereditary angioedema; GI, gastrointestinal; GU, genitourinary; MI, myocardial infarction; MRI, magnetic resonance imaging; NA, not applicable; OD, once daily; PD, pharmacodynamics; PK, pharmacokinetics.
Study recruitment status as of July 2022.