{ "data": { "asOf": "2026-10-30T22:24:36.521Z", "dedupMethod": "exact cross-registry identifier matching; probabilistic = review only", "byRegistry": { "clinicaltrials_gov": 1 }, "canonicalTrial": { "canonicalId": "NCT07080905", "title": "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", "scientificTitle": "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", "status": "recruiting", "studyType": "interventional", "phase": [ "PHASE3" ], "conditions": [ "Hemophilia B" ], "interventions": [ { "name": "CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)", "type": "GENETIC", "description": "Administered as a single IV infusion." } ], "sponsor": { "name": "CSL Behring", "type": "INDUSTRY" }, "locations": [ { "city": "Orange", "country": "United States", "facility": "Center for Inherited Blood Disorders" }, { "city": "San Diego", "country": "United States", "facility": "Rady Children's Hospital San Diego" }, { "city": "Gainesville", "country": "United States", "facility": "University of Florida" }, { "city": "Atlanta", "country": "United States", "facility": "Arthur M. Blank Hospital - Children's Healthcare of Atlanta" }, { "city": "Ann Arbor", "country": "United States", "facility": "University of Michigan Medical Center" }, { "city": "Memphis", "country": "United States", "facility": "St. Jude Children's Research Hospital" }, { "city": "Westmead", "country": "Australia", "facility": "The Children's Hospital at Westmead" }, { "city": "Brisbane", "country": "Australia", "facility": "Queensland Children's Hospital" }, { "city": "Vienna", "country": "Austria", "facility": "Medical University Vienna" }, { "city": "Leuven", "country": "Belgium", "facility": "UZ Leuven - Centrum voor Moleculaire en Vasculaire Biologie" }, { "city": "Lille", "country": "France", "facility": "Lille University Hospital Center - Coeur Poumon Institute" }, { "city": "Marseille", "country": "France", "facility": "CHU Hopital de la Timone" }, { "city": "Ramat Gan", "country": "Israel", "facility": "Chaim Sheba Medical Center" }, { "city": "Barcelona", "country": "Spain", "facility": "Hospital Sant Joan de Déu" }, { "city": "Madrid", "country": "Spain", "facility": "La Paz University Hospital" }, { "city": "London", "country": "United Kingdom", "facility": "St Thomas Hospital" }, { "city": "Oxford", "country": "United Kingdom", "facility": "John Radcliffe Hospital - Oxford University Hospitals NHS" } ], "outcomes": { "other": [], "primary": [ "Annualized Bleeding Rate (ABR)" ], "secondary": [ "Canadian Hemophilia Outcomes-Kids Life Assessment Tool (CHO-KLAT) total score and change from baseline", "Endogenous FIX activity", "Change from baseline in endogenous FIX activity", "Annualized consumption of FIX replacement therapy", "Annualized infusion rate of FIX replacement therapy", "Number of participants remaining free of continuous FIX prophylaxis", "Percentage of participants remaining free of continuous FIX prophylaxis", "ABR for spontaneous, joint, and FIX-treated bleeding episodes", "Correlation of FIX activity levels and pre-CSL222 AAV5 NAb titer", "Number of new target joints and resolution of preexisting target joints", "Number of participants with zero bleeds and zero FIX-treated bleeds", "Percentage of participants with zero bleeds and zero FIX-treated bleeds", "Change in the EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Visual Analogue Scale (VAS) Overall Score", "Change in the EQ-5D-5L Index Scores", "Change in the Patient-Reported Outcomes Measurement Information System (PROMIS)-25 total score", "Change from baseline in PROMIS-29 total score", "Number of participants with endogenous FIX activity of ≥ 5%", "Percentage of participants with endogenous FIX activity of ≥ 5%", "Adverse events - number of participants", "Adverse events - percentage of participants", "Adverse events - number of events", "Change in liver ultrasound", "Number of participants with antibodies against AAV5", "Number of participants who develop FIX inhibitors", "Number of clinically significant clinical laboratory tests (Hematology and Biochemistry) reported as an AE", "Number of participants with clinically significant ALT/AST levels", "Percentage of participants with clinically significant ALT/AST levels", "Number of participants using corticosteroid for change in AST/ALT levels", "Duration of corticosteroid use for change in AST/ALT levels", "Mean inflammatory markers values", "Change from baseline in Inflammatory markers" ] }, "eligibility": { "sex": "MALE", "criteria": "Inclusion Criteria:\n\n* Key Inclusion Criteria for the Lead-in Period: Assigned male sex at birth\n* 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.\n* 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.\n* On stable continuous FIX prophylaxis for at least 2 months before Screening.\n* Minimum of 75 previous exposure days of treatment with FIX protein before Screening.\n* Additional Key Inclusion Criteria for the Treatment Period:\n\nCompleted the Lead-in Period: minimum of 6 months (26 weeks) of lead-in data collected and eligibility has been confirmed.\n\n* Aged ≥ 12 to \\< 18 years at the time of CSL222 treatment.\n\nExclusion Criteria:\n\n* Key Exclusion Criteria for the Lead-in Period: History of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory results).\n* Screening laboratory values (based on central laboratory results):\n\n * Total bilirubin \\> 2 × the upper limit of normal (ULN).\n * Alanine aminotransferase (ALT) \\> 2 × the ULN.\n * Aspartate aminotransferase (AST) \\> 2 × the ULN.\n * Alkaline phosphatase (ALP) \\> 2 × the ULN.\n * Serum creatinine \\> 2 × the ULN.\n * Hemoglobin \\< 8 g/dL.\n* Any condition other than hemophilia B resulting in an increased bleeding tendency.\n* Thrombocytopenia, defined as a platelet count below 50 × 10\\^9/L, at screening (based on central laboratory results).\n* 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.\n* Additional Key Exclusion Criteria for the Treatment Period: Positive FIX inhibitor test at Visit L-Final (based on central laboratory results)\n* AAV5 NAb titer \\> 1:900 as assessed at Visit LX (last visit before Visit L-Final).\n* Visit L-Final laboratory values (based on central laboratory results) of:\n\n * Total bilirubin \\> 2 × the ULN\n * ALT \\> 2 × the ULN.\n * AST 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