Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies
CSL Behring
Summary
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Considered legally an adult, as defined by country regulations. * Has congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to \[\<=\] 2% of normal circulating FIX) for which the participant is on continuous routine FIX prophylaxis. * Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results). * Has greater than (\>) 150 previous exposure days to FIX replacement therapy. * Has been on stable FIX prophylaxis for at least 2 months…
Interventions
- GeneticCSL222 (AAV5-hFIXco-Padua)
Administered as a single IV infusion.
Locations (26)
- University of California, San Diego (UCSD)San Diego, California
- University of MichiganAnn Arbor, Michigan
- Royal Prince Alfred HospitalCamperdown, New South Wales
- Royal Brisbane HospitalHerston, Queensland
- Royal Adelaide HospitalAdelaide, South Australia
- The Alfred HospitalMelbourne, Victoria