The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).
Study Type
OBSERVATIONAL
Enrollment
56
Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).
Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Percentage of Participants With SAEs and AESIs
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Number of SAEs and AESIs
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Annualized Bleeding Rate (ABR)
The total, spontaneous, joint, and traumatic bleeds will be analyzed.
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Endogenous Factor IX (FIX) Activity
Endogenous FIX activity (expressed as percentage \[%\]) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay.
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Change from Baseline by Visit in Endogenous FIX Activity
Endogenous FIX activity (expressed as %) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay.
Time frame: At Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
Annualized Consumption of FIX Replacement Therapy
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10-15 Phoenix Childrens Hospital
Phoenix, Arizona, United States
10-14 Arkansas Children's Hospital - Pharmacology
Little Rock, Arkansas, United States
10-22 Orthopaedic Institute for Children
Los Angeles, California, United States
10-21 Children's Hospital of Los Angeles
Los Angeles, California, United States
10-63 UC Davis Medical Center
Sacramento, California, United States
10-12 University of California, San Diego (UCSD)
San Diego, California, United States
10-25 University of Colorado Denver
Aurora, Colorado, United States
10-19 University of South Florida
Tampa, Florida, United States
10-10 University of Michigan Medical Center
Ann Arbor, Michigan, United States
10-16 University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
...and 18 more locations
The annualized consumption of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided.
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Annualized Infusion Rate of FIX Replacement Therapy
Annualized infusion rate of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided.
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Number of Participants Remaining Free of Continuous FIX Prophylaxis
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Percentage of Participants Remaining Free of Continuous FIX Prophylaxis
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Number of New Target Joints
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Percentage Resolution of Pre-existing Target Joints
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value
The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D visual analogue scale (VAS). The EQ-5D-5L descriptive system of health-related quality of life (QoL) consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls \[Hernández Alava et al, 2023\], using the United Kingdom value set.
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Change From Baseline in EQ-5D-5L Index Value
The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ-5D VAS. The EQ-5D-5L descriptive system of health-related QoL consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls \[Hernández Alava et al, 2023\], using the United Kingdom value set.
Time frame: Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
EQ-5D VAS Score
The EQ-5D VAS, which measures overall health status on a vertical VAS, ranges from 0 to 100. A higher score indicates better QoL.
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Change From Baseline in EQ-5D VAS Score
The EQ-5D VAS, which measures overall health status on a vertical VAS, ranges from 0 to 100. A higher score indicates better QoL.
Time frame: Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score
The Hem-A-QoL consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The total score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflecting a better QoL.
Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15
Change From Baseline in Hem-A-QoL Total Score
The Hem-A-QoL consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The total score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflecting a better QoL.
Time frame: Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15