NCT07080905PHASE3Recruiting
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B
CSL Behring
Start Date
7/28/2025
Completion Date
10/24/2033
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 Criteria
Age Range: No minimum to No maximum
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 Screening.
* Additional Key Inclusion Criteria for the Treatment Period:
Completed the Lead-in Period: minimum of 6 months (26 weeks) of lead-in data collected and eligibility has been confirmed.
* Aged ≥ 12 to \< 18 years at the time of CSL222 treatment.
Exclusion Criteria:
* Key Exclusion Criteria for the Lead-in Period:
History of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory results).
* Screening laboratory values (based on central laboratory results):
* Total bilirubin \> 2 × the upper limit of normal (ULN).
* Alanine aminotransferase (ALT) \> 2 × the ULN.
* Aspartate aminotransferase (AST) \> 2 × the ULN.
* Alkaline phosphatase (ALP) \> 2 × the ULN.
* Serum creatinine \> 2 × the ULN.
* Hemoglobin \< 8 g/dL.
* Any condition other than hemophilia B resulting in an increased bleeding tendency.
* Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at screening (based on central laboratory results).
* Any uncontrolled or untreated infection (human immunodeficiency virus, hepatitis C, etc) or any other significant concurrent, uncontrolled medical condition, as evaluated by the investigator, including, but not limited to renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral, or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder evaluated by the investigator to interfere with adherence to the Clinical Study Protocol procedures or with the degree of tolerance to CSL222.
* Additional Key Exclusion Criteria for the Treatment Period:
Positive FIX inhibitor test at Visit L-Final (based on central laboratory results)
* AAV5 NAb titer \> 1:900 as assessed at Visit LX (last visit before Visit L-Final).
* Visit L-Final laboratory values (based on central laboratory results) of:
* Total bilirubin \> 2 × the ULN
* ALT \> 2 × the ULN.
* AST \> 2 × the ULN.
* ALP \> 2 × the ULN.
* Serum creatinine \> 2 × the ULN.
* Hemoglobin \< 8 g/dL.
* Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at Visit L-Final (based on central laboratory results).
Interventions
GENETIC
CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
Conditions
Hemophilia B
Locations
Center for Inherited Blood Disorders
Orange, California 92868
United States
University of Florida
Gainesville, Florida 32611
United States
Arthur M. Blank Hospital - Children's Healthcare of Atlanta
Atlanta, Georgia 30329
United States
University of Michigan Medical Center
Ann Arbor, Michigan 48109
United States
St. Jude Children's Research Hospital
Memphis, Tennessee 38105
United States
Medical University Vienna
Vienna, 1090
Austria
UZ Leuven - Centrum voor Moleculaire en Vasculaire Biologie
Leuven, 3000
Belgium
Chaim Sheba Medical Center
Ramat Gan, 5262000
Israel
Hospital Sant Joan de Déu
Barcelona, 08950
Spain
La Paz University Hospital
Madrid, 28046
Spain
St Thomas Hospital
London, SE1 7EH
United Kingdom
John Radcliffe Hospital - Oxford University Hospitals NHS
Oxford, OX3 9DU
United Kingdom