Table 1. Summary of valoctocogene... | American Society of Hematology

Table 1.

Summary of valoctocogene roxaparvovec clinical trials

Name	Phase	Dose (vg/kg)	Unique criteria	N	Immunosuppression	Outcomes	Trial status	NCT#	References
BMN270-201	1/2	6 × 10¹²	See text	1	None	See text	Active, not recruiting	02576795	3,4,7
		2 × 10¹³		1	None
		6 × 10¹³		7	7/7 (prophylactic)
		4 × 10¹³		6	4/6 on-demand
BMN270-203	1/2	6 × 10¹³	Positive AAV Ab	1	-	No available data	Active, not recruiting	03520712	-
BMN270-205 (GENEr8-INH)	1/2	6 × 10¹³	Active or prior FVIII Inhibitors	4	4/4	N = 2 active inhibitors, 1 amnestic response, 1 sign of tolerance; N = 2 prior inhibitors and did not develop inhibitors	Recruiting	04684940	³⁵
GENEr8-1	3	6 × 10¹³	See text	134	106/134	See text	Active, not recruiting	03370913	5,6
GENEr8-2	3	4 × 10¹³	See text	1	1/1	See text	Completed	03392974	³⁶
GENEr8-3	3	6 × 10¹³	Prophylactic steroids	22	40 mg per day steroids for 8 weeks; taper dose for 11 weeks	>90% participants experienced ALT elevations; FVIII 14.5% at year-1	Active, not recruiting	04323098	³⁷

Name	Phase	Dose (vg/kg)	Unique criteria	N	Immunosuppression	Outcomes	Trial status	NCT#	References
BMN270-201	1/2	6 × 10¹²	See text	1	None	See text	Active, not recruiting	02576795	3,4,7
		2 × 10¹³		1	None
		6 × 10¹³		7	7/7 (prophylactic)
		4 × 10¹³		6	4/6 on-demand
BMN270-203	1/2	6 × 10¹³	Positive AAV Ab	1	-	No available data	Active, not recruiting	03520712	-
BMN270-205 (GENEr8-INH)	1/2	6 × 10¹³	Active or prior FVIII Inhibitors	4	4/4	N = 2 active inhibitors, 1 amnestic response, 1 sign of tolerance; N = 2 prior inhibitors and did not develop inhibitors	Recruiting	04684940	³⁵
GENEr8-1	3	6 × 10¹³	See text	134	106/134	See text	Active, not recruiting	03370913	5,6
GENEr8-2	3	4 × 10¹³	See text	1	1/1	See text	Completed	03392974	³⁶
GENEr8-3	3	6 × 10¹³	Prophylactic steroids	22	40 mg per day steroids for 8 weeks; taper dose for 11 weeks	>90% participants experienced ALT elevations; FVIII 14.5% at year-1	Active, not recruiting	04323098	³⁷

Data are current as of March 2024.

Ab, antibody.