An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects with Hemophilia B

Conditions

Hemophilia B

Brief summary

Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs), Percentage of Participants with SAEs and AESIs, Number of SAEs and AESIs

Detailed description

Annualized Bleeding Rate (ABR), Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes, Endogenous Factor IX (FIX) Activity, Change from Baseline by Visit in Endogenous FIX Activity, Annualized Consumption of FIX Replacement Therapy, Annualized Infusion Rate of FIX Replacement Therapy, Number of Participants Remaining Free of Continuous FIX Prophylaxis, Percentage of Participants Remaining Free of Continuous FIX Prophylaxis, Number of New Target Joints, Percentage Resolution of Pre-existing Target Joints, EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value, Change From Baseline in EQ-5D-5L Index Value, EQ-5D VAS Score, Change From Baseline in EQ-5D VAS Score, Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score, Change From Baseline in Hem-A-QoL Total Score

Related papers

No related papers found yet.

Interventions

DRUGHemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion

Eligibility

Sex/Gender

Male

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs), Percentage of Participants with SAEs and AESIs, Number of SAEs and AESIs	—

Secondary

Measure	Time frame
Annualized Bleeding Rate (ABR), Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes, Endogenous Factor IX (FIX) Activity, Change from Baseline by Visit in Endogenous FIX Activity, Annualized Consumption of FIX Replacement Therapy, Annualized Infusion Rate of FIX Replacement Therapy, Number of Participants Remaining Free of Continuous FIX Prophylaxis, Percentage of Participants Remaining Free of Continuous FIX Prophylaxis, Number of New Target Joints, Percentage Resolution of Pre-existing Target Joints, EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value, Change From Baseline in EQ-5D-5L Index Value, EQ-5D VAS Score, Change From Baseline in EQ-5D VAS Score, Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score, Change From Baseline in Hem-A-QoL Total Score	—

Measure

Time frame

Annualized Bleeding Rate (ABR), Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes, Endogenous Factor IX (FIX) Activity, Change from Baseline by Visit in Endogenous FIX Activity, Annualized Consumption of FIX Replacement Therapy, Annualized Infusion Rate of FIX Replacement Therapy, Number of Participants Remaining Free of Continuous FIX Prophylaxis, Percentage of Participants Remaining Free of Continuous FIX Prophylaxis, Number of New Target Joints, Percentage Resolution of Pre-existing Target Joints, EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value, Change From Baseline in EQ-5D-5L Index Value, EQ-5D VAS Score, Change From Baseline in EQ-5D VAS Score, Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score, Change From Baseline in Hem-A-QoL Total Score

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Countries

Belgium, Denmark, Germany, Ireland, Netherlands, Sweden

Outcome results

None listed