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

Purpose

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.

Condition

  • Hemophilia B

Eligibility

Eligible Ages
Between 138 Months and 206 Months
Eligible Sex
Male
Accepts 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 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).

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
CSL222 		Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10^13 genome copies per kilogram of body weight (gc/kg).
  • Genetic: CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)
    Administered as a single IV infusion.
    Other names:
    • Etranacogene Dezaparvovec

Recruiting Locations

University of Florida
Gainesville 4156404, Florida 4155751 32611
Contact:
Trial Contact

Arthur M. Blank Hospital - Children's Healthcare of Atlanta
Atlanta 4180439, Georgia 4197000 30329
Contact:
Trial Contact

University of Michigan Medical Center
Ann Arbor 4984247, Michigan 5001836 48109
Contact:
Trial Contact

St. Jude Children's Research Hospital
Memphis 4641239, Tennessee 4662168 38105
Contact:
Trial Contact

More Details

Status
Recruiting
Sponsor
CSL Behring

Study Contact

Trial Registration Coordinator
1-610-878-4697
clinicaltrials@cslbehring.com