BridgeMD
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
EU CTIS ID: 2023-505805-18-00
What this study is testing
The objective of this study is to investigate the efficacy, safety, and tolerability of CSL222 (International Nonproprietary Name: etranacogene dezaparvovec, previously termed AMT 061) administered to adolescent male subjects (≥ 12 to < 18 years of age) with severe or moderately severe hemophilia B.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Assigned male sex at birth.
- 2. 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.
- 3. 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.
- 4. On stable FIX continuous prophylaxis for at least 2 months before Screening.
- 5. Minimum of 75 previous exposure days of treatment with FIX protein before Screening.
- 6. 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.
You likely can't join if
- 1. History of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory results).
- 2. Screening laboratory values (based on central laboratory results): ◦ Total bilirubin greater than (>) 2 x the upper limit of normal (ULN) ◦ ALT > 2 x the ULN. ◦ AST > 2 x the ULN. ◦ ALP > 2 x the ULN. ◦ Serum creatinine > 2 x the ULN. ◦ Hemoglobin < 8 g/dL.
- 3. Any condition other than hemophilia B resulting in an increased bleeding tendency.
- 4. Thrombocytopenia, defined as a platelet count below 50 x 10^9/Liter, at Screening (based on central laboratory results).
- 5. 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.
- 6. Additional Key Exclusion Criteria for the Treatment Period: Positive FIX inhibitor test at Visit L-Final (based on central laboratory results).
See the full eligibility criteria
- 1. Assigned male sex at birth.
- 2. 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.
- 3. 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.
- 4. On stable FIX continuous prophylaxis for at least 2 months before Screening.
- 5. Minimum of 75 previous exposure days of treatment with FIX protein before Screening.
- 6. 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.
- 7. Additional Key Inclusion Criteria for the Treatment Period: Aged >= 12 to < 18 years at the time of CSL222 treatment.
- 1. History of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory results).
- 2. Screening laboratory values (based on central laboratory results): ◦ Total bilirubin greater than (>) 2 x the upper limit of normal (ULN) ◦ ALT > 2 x the ULN. ◦ AST > 2 x the ULN. ◦ ALP > 2 x the ULN. ◦ Serum creatinine > 2 x the ULN. ◦ Hemoglobin < 8 g/dL.
- 3. Any condition other than hemophilia B resulting in an increased bleeding tendency.
- 4. Thrombocytopenia, defined as a platelet count below 50 x 10^9/Liter, at Screening (based on central laboratory results).
- 5. 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.
- 6. Additional Key Exclusion Criteria for the Treatment Period: Positive FIX inhibitor test at Visit L-Final (based on central laboratory results).
- 7. Additional Key Exclusion Criteria for the Treatment Period: AAV5 NAb titer > 1:900 as assessed at Visit LX (last visit before Visit L-Final).
- 8. Additional Key Exclusion Criteria for the Treatment Period: 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.
- 9. Additional Key Exclusion Criteria for the Treatment Period: thrombocytopenia, defined as a platelet count below 50 × 10^9/L, at Visit L-Final (based on central laboratory results).
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- Australia
- United States
- Israel
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; Australia; United States; Israel. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.