Table 2. Phase 3 study of etranacogene... | American Society of Hematology

Table 2.

Phase 3 study of etranacogene dezaparvovec

Current sponsor Original sponsor	CSL Behring uniQure biopharma B.V.
Vector (alternative name) and dose	AAV5-hFIXco-Padua (AMT-061) 2 × 10¹³ gc/kg of body weight
Approval status	Approved in United States: 22 November 2022
	Approved in European Union: 20 February 2023
Indication	Indicated for treatment of adults with hemophilia B who:
	Currently use FIX prophylaxis therapy, or
	Have current or historical life-threatening hemorrhage, or
	Have repeated, serious spontaneous bleeding episodes
Trial	HOPE-B (NCT03569891)
	54 participants
	Multinational, open-label, single-arm
Inclusion criteria	Adult males
	Severe or moderately severe hemophilia B (FIX ≤ 2 IU/dL)
	Currently on FIX prophylaxis
	>150 previous exposure days of treatment with FIX protein
	With or without preexisting NAbs to AAV5
Selected exclusion criteria	FIX inhibitors before or at screening
	Select screening laboratory value >2 times upper limit of normal
	Positive HIV test at screening, not controlled with antiviral therapy
	Active infection with hepatitis B or C virus at screening
	Previous gene therapy treatment
	Receipt of an experimental agent within 60 d before screening
	Current participation or anticipated participation within 1 y after study drug administration in this trial in any other interventional clinical trial involving drugs or devices

Current sponsor Original sponsor	CSL Behring uniQure biopharma B.V.
Vector (alternative name) and dose	AAV5-hFIXco-Padua (AMT-061) 2 × 10¹³ gc/kg of body weight
Approval status	Approved in United States: 22 November 2022
	Approved in European Union: 20 February 2023
Indication	Indicated for treatment of adults with hemophilia B who:
	Currently use FIX prophylaxis therapy, or
	Have current or historical life-threatening hemorrhage, or
	Have repeated, serious spontaneous bleeding episodes
Trial	HOPE-B (NCT03569891)
	54 participants
	Multinational, open-label, single-arm
Inclusion criteria	Adult males
	Severe or moderately severe hemophilia B (FIX ≤ 2 IU/dL)
	Currently on FIX prophylaxis
	>150 previous exposure days of treatment with FIX protein
	With or without preexisting NAbs to AAV5
Selected exclusion criteria	FIX inhibitors before or at screening
	Select screening laboratory value >2 times upper limit of normal
	Positive HIV test at screening, not controlled with antiviral therapy
	Active infection with hepatitis B or C virus at screening
	Previous gene therapy treatment
	Receipt of an experimental agent within 60 d before screening
	Current participation or anticipated participation within 1 y after study drug administration in this trial in any other interventional clinical trial involving drugs or devices