Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
CSL Behring
Summary
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.
Eligibility
- Age range
- 11–17 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: * Key Inclusion Criteria for the Lead-in Period: Assigned male sex at birth * Aged ≥ 138 months (11 years and 6 months) to less than (\<) 206 months (17 years and 2 months) at the time of informed consent / assent. * 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 has been on continuous FIX prophylaxis. * On stable continuous FIX prophylaxis for at least 2 months before Screening. * Minimum of 75 previous exposure days of treatment with FIX protein before Sc…
Interventions
- GeneticCSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
Administered as a single IV infusion.
Locations (2)
- University of Florida - 84000399Gainesville, Florida
- University of Michigan Medical Center - 84000491Ann Arbor, Michigan