Table 2.

Clinical trials of novel alternative hemostatic agents for hemophilia

Novel AgentNCTSponsorRouteSubjectStatus
1. Coagulation factor mutants or mimics 
 ACE910 NCT02622321 Hoffmann-La Roche SQ HA/I Phase 1/2 ongoing 
 NCT02847637    Phase 3 planned 
 Super FVa NA Bayer IV — Preclinical ongoing 
 FXaI16→L NCT01897142 Pfizer IV HA/I Phase 1 ongoing 
 FVIII-∆P/F NA Avelas IV; AAV — Preclinical ongoing 
2. Natural anticoagulant knock-down or disruption 
 ALN-AT NCT02554773 Alnylam SQ HA/HB/I Phase 1, 2 ongoing 
 NCT03001830  SQ  Phase 3 planned 
 Anti-TFPI NCT02540187 Bayer IV, SQ HA/HB/I Phase 1 ongoing 
 NCT02571569    Phase 2, 3 planned 
 Anti-TFPI NCT015555749 Novo Nordisk IV, SQ HA/HB Phase 1 ongoing 
 NCT02490787    Phase 2, 3 planned 
 Anti-TFPI NCT02540187 Pfizer IV, SQ HA/HB Phase 1 ongoing 
 NCT02974855    Phase 2, 3 planned 
 KRK α1AT — ApcinteX IV — Phase 1 planned 
Novel AgentNCTSponsorRouteSubjectStatus
1. Coagulation factor mutants or mimics 
 ACE910 NCT02622321 Hoffmann-La Roche SQ HA/I Phase 1/2 ongoing 
 NCT02847637    Phase 3 planned 
 Super FVa NA Bayer IV — Preclinical ongoing 
 FXaI16→L NCT01897142 Pfizer IV HA/I Phase 1 ongoing 
 FVIII-∆P/F NA Avelas IV; AAV — Preclinical ongoing 
2. Natural anticoagulant knock-down or disruption 
 ALN-AT NCT02554773 Alnylam SQ HA/HB/I Phase 1, 2 ongoing 
 NCT03001830  SQ  Phase 3 planned 
 Anti-TFPI NCT02540187 Bayer IV, SQ HA/HB/I Phase 1 ongoing 
 NCT02571569    Phase 2, 3 planned 
 Anti-TFPI NCT015555749 Novo Nordisk IV, SQ HA/HB Phase 1 ongoing 
 NCT02490787    Phase 2, 3 planned 
 Anti-TFPI NCT02540187 Pfizer IV, SQ HA/HB Phase 1 ongoing 
 NCT02974855    Phase 2, 3 planned 
 KRK α1AT — ApcinteX IV — Phase 1 planned 

NCT indicates clinicaltrials.gov number.

AAV, adeno-associated virus; HA, hemophilia A; HB, hemophilia B; I, inhibitor; NA, not available; SQ, subcutaneous.

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