Table 1. Novel agents targeting adhesion... | American Society of Hematology

Table 1.

Novel agents targeting adhesion

Name	Mechanism	Trial phase, type, NCT#	Primary end points	Status	Results and comments
Rivipansel (GMI-1070)*	Small-molecule pan-selectin antagonist; maximal activity against E-selectin	1/2, single arm open label, NCT00911495	Safety	Completed	Well tolerated; increase in total WBC, without associated symptoms⁷⁶
		2, RDBPC, NCT01119833	Time to resolution of VOC	Completed	Trend in expected direction in primary end point
		2, RDBPC, NCT01119833	Time to resolution of VOC	Completed	Cumulative opioid use significantly decreased (by 83%; P = .010)¹⁰
		3, RDBPC, NCT02187003	Time to readiness for discharge	Recruiting	All SCD genotypes; age ≥6 y
		3, RDBPC, NCT02187003	Time to readiness for discharge	Recruiting	IV infusion every 12 h during VOC
SEG101 (crizanlizumab)	Humanized mAb against P-selectin	2, RDBPC, NCT1895361	Annual rate of sickle cell–related pain crises with high-dose crizanlizumab vs placebo	Completed	All SCD genotypes; reduction in pain crisis rate by 45% in high-dose arm; improvement in various secondary end points; no improvement in markers of hemolysis⁷⁷
		2, single arm open label, NCT03264989	PKs and PDs	Recruiting	All SCD genotypes
		2, single arm open label, NCT03474965	PKs and PDs	Not yet recruiting	All SCD genotypes; age 6 mo to 17 y
IVIG*	Disrupts neutrophil-mediated sRBC capture	1, RDBPC, NCT01757418	Safety, dose finding, and neutrophil activation	Completed	Low-dose IVIG was safe, well tolerated, and stabilized Mac-1 activity¹⁴
IVIG*	Disrupts neutrophil-mediated sRBC capture	2, RDBPC, NCT01757418	Length of VOC	Recruiting	Single low-dose during VOC
Sevuparin*	Heparinoid with anti–P- and anti–L-selectin activity	2, RDBPC, NCT02515838	Time to resolution of VOC	Recruiting	All SCD genotypes; continuous IV infusion

Name	Mechanism	Trial phase, type, NCT#	Primary end points	Status	Results and comments
Rivipansel (GMI-1070)*	Small-molecule pan-selectin antagonist; maximal activity against E-selectin	1/2, single arm open label, NCT00911495	Safety	Completed	Well tolerated; increase in total WBC, without associated symptoms⁷⁶
		2, RDBPC, NCT01119833	Time to resolution of VOC	Completed	Trend in expected direction in primary end point
		2, RDBPC, NCT01119833	Time to resolution of VOC	Completed	Cumulative opioid use significantly decreased (by 83%; P = .010)¹⁰
		3, RDBPC, NCT02187003	Time to readiness for discharge	Recruiting	All SCD genotypes; age ≥6 y
		3, RDBPC, NCT02187003	Time to readiness for discharge	Recruiting	IV infusion every 12 h during VOC
SEG101 (crizanlizumab)	Humanized mAb against P-selectin	2, RDBPC, NCT1895361	Annual rate of sickle cell–related pain crises with high-dose crizanlizumab vs placebo	Completed	All SCD genotypes; reduction in pain crisis rate by 45% in high-dose arm; improvement in various secondary end points; no improvement in markers of hemolysis⁷⁷
		2, single arm open label, NCT03264989	PKs and PDs	Recruiting	All SCD genotypes
		2, single arm open label, NCT03474965	PKs and PDs	Not yet recruiting	All SCD genotypes; age 6 mo to 17 y
IVIG*	Disrupts neutrophil-mediated sRBC capture	1, RDBPC, NCT01757418	Safety, dose finding, and neutrophil activation	Completed	Low-dose IVIG was safe, well tolerated, and stabilized Mac-1 activity¹⁴
IVIG*	Disrupts neutrophil-mediated sRBC capture	2, RDBPC, NCT01757418	Length of VOC	Recruiting	Single low-dose during VOC
Sevuparin*	Heparinoid with anti–P- and anti–L-selectin activity	2, RDBPC, NCT02515838	Time to resolution of VOC	Recruiting	All SCD genotypes; continuous IV infusion

Unless otherwise specified, studies included only adult patients with HbSS or HbSβ0.

mAb, monoclonal antibody; PD, pharmacodynamic; RDBPC, randomized double blind placebo controlled; WBC, white blood cell count.

Study drug administered during acute VOC.

View Large

This Feature Is Available To Subscribers Only