Skip to main content

CSL222_3004

  • Status
    Accepting Candidates
  • Age
    138 Months - 206 Months
  • Sexes
    Male
  • Healthy Volunteers
    No
I'm interested
Share this study

Objective

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.

Details

Full study title Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (&#8805; 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B
Protocol number OCR45176
ClinicalTrials.gov ID NCT07080905
Phase Phase 3

Eligibility

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 ( 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).

Lead researcher

  • Tung T Wynn, MD
    Pediatric Hematologist/Oncologist (Child Cancer Specialist)
    Tung T Wynn

Participate in a study

Here are some general steps to consider when participating in a research study:

  1. Step
    1

    Contact the research team

    Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.

    Primary contact

  2. Step
    2

    Get screened to confirm eligibility

    You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.

  3. Step
    3

    Provide your consent to participate

    If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.

  4. Step
    4

    Participate

    If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.