Table 4.

Complement inhibitors under clinical investigation for treatment of HSCT-TMA

Drug	Target/mechanism of action	Class	Company	Status	ClinicalTrials.gov
Eculizumab	C5 inhibition	mAb	Alexion Pharmaceuticals	Phase 2 ongoing; off-label use in clinic [161]	NCT03518203 (pediatric + adult) [162]
Ravulizumab (ALXN1210)	C5 inhibition	mAb	Alexion Pharmaceuticals	Phase 3 ongoing	NCT04543591 (adolescent + adult) [141] NCT04557735 (pediatric) [142]
Nomacopan (Coversin)	C5 and LTB4 inhibition	Recombinant protein	Akari Therapeutics	Phase 3 ongoing	NCT04784455 (pediatric) [145]
Narsoplimab (OMS721)	MASP-2 inhibition	mAb	Omeros Corporation	Phase 2 complete	NCT02222545 (adult) [147]

HSCT-TMA Hematopoietic stem cell transplantation-associated thrombotic microangiopathy, LTB4 Leukotriene B4, mAb Monoclonal antibody, MASP-2 Mannan-binding lectin-associated serine protease 2