Hemophilia B
Conditions
Brief summary
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).
Interventions
GENETICAAV5-hFIXco-Padua
Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).
Sponsors
CSL Behring
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Received treatment with CSL222 in Study CSL222\_2001 or Study CSL222\_3001, and completed participation in Study CSL222\_2001 or Study CSL222\_3001 or at least 5 years have passed since receiving CSL222.
Exclusion criteria
Not Applicable
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Percentage of Participants With SAEs and AESIs | From Year 5 after administration of CSL222 in parent study up to Year 15 |
| Number of SAEs and AESIs | From Year 5 after administration of CSL222 in parent study up to Year 15 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annualized Bleeding Rate (ABR) | From Year 5 after administration of CSL222 in parent study up to Year 15 | The total, spontaneous, joint, and traumatic bleeds will be analyzed. |
| Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes | From Year 5 after administration of CSL222 in parent study up to Year 15 | — |
| Endogenous Factor IX (FIX) Activity | From Year 5 after administration of CSL222 in parent study up to Year 15 | Endogenous FIX activity (expressed as percentage \[%\]) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay. |
| Change from Baseline by Visit in Endogenous FIX Activity | At Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 | Endogenous FIX activity (expressed as %) will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay. |
| Annualized Consumption of FIX Replacement Therapy | From Year 5 after administration of CSL222 in parent study up to Year 15 | The annualized consumption of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided. |
| Annualized Infusion Rate of FIX Replacement Therapy | From Year 5 after administration of CSL222 in parent study up to Year 15 | Annualized infusion rate of FIX replacement therapy (excluding FIX replacement for invasive procedures) will be provided. |
| Number of Participants Remaining Free of Continuous FIX Prophylaxis | From Year 5 after administration of CSL222 in parent study up to Year 15 | — |
| Percentage of Participants Remaining Free of Continuous FIX Prophylaxis | From Year 5 after administration of CSL222 in parent study up to Year 15 | — |
| Number of New Target Joints | From Year 5 after administration of CSL222 in parent study up to Year 15 | — |
| Percentage Resolution of Pre-existing Target Joints | From Year 5 after administration of CSL222 in parent study up to Year 15 | — |
| EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value | From Year 5 after administration of CSL222 in parent study up to Year 15 | 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. |
| Change From Baseline in EQ-5D-5L Index Value | Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 | 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. |
| Change From Baseline in EQ-5D VAS Score | Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 | The EQ-5D VAS, which measures overall health status on a vertical VAS, ranges from 0 to 100. A higher score indicates better QoL. |
| EQ-5D VAS Score | From Year 5 after administration of CSL222 in parent study up to Year 15 | The EQ-5D VAS, which measures overall health status on a vertical VAS, ranges from 0 to 100. A higher score indicates better QoL. |
| Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score | From Year 5 after administration of CSL222 in parent study up to Year 15 | 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. |
| Change From Baseline in Hem-A-QoL Total Score | Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15 | 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. |
Countries
Belgium, Denmark, Germany, Ireland, Netherlands, Sweden, United Kingdom, United States
Outcome results
None listed