Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
- Conditions
- Hemophilia B
- Interventions
- Genetic: AAV5-hFIXco-Padua
- Registration Number
- NCT05962398
- Lead Sponsor
- CSL Behring
- Brief Summary
The primary purpose of this study is to assess the long-term safety in male adults with hemophilia B who were treated with CSL222 in studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 56
- Treatment with CSL222 in Study CSL222_2001 or Study CSL222_3001.
- Provided written informed consent.
- Willing and able to adhere to all protocol requirements.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description CSL222 AAV5-hFIXco-Padua Participants who received CSL222 and completed either the study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891) will be followed up from Year 5 after administration of CSL222 in parent study up to Year 15.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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.
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.
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.
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.
Trial Locations
- Locations (20)
10-25 University of Colorado Denver
🇺🇸Aurora, Colorado, United States
10-16 University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
10-20 Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
11-30 UZ Leuven - Campus Gasthuisberg
🇧🇪Leuven, Belgium
12-32 Rigshospitalet
🇩🇰Copenhagen, Denmark
13-34 Erasmus University Medical Center
🇳🇱Rotterdam, Netherlands
13-36 Universitair Medisch Centrum Utrecht
🇳🇱Utrecht, Netherlands
Phoenix Childrens Hospital
🇺🇸Phoenix, Arizona, United States
Arkansas Children's Hospital - Pharmacology
🇺🇸Little Rock, Arkansas, United States
Orthopaedic Institute for Children
🇺🇸Los Angeles, California, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
University of California, San Diego (UCSD)
🇺🇸San Diego, California, United States
University of South Florida
🇺🇸Tampa, Florida, United States
University of Michigan Medical Center
🇺🇸Ann Arbor, Michigan, United States
Cliniques Universitaires Saint-Luc
🇧🇪Brussels, Belgium
Universitair Ziekenhuis Leuven
🇧🇪Leuven, Belgium
Amsterdam Universitair Medische Centra (UMC)
🇳🇱Amsterdam Zuidoost, Netherlands
Barts Health NHS Trust
🇬🇧London, England, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, United Kingdom