Hemophilia B study: CSL222
Official 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
ClinicalTrials.gov ID: NCT07080905
What this study is testing
What is CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)?
CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua) is an investigational medicine, given as an IV infusion (through a vein), being studied as a potential treatment for hemophilia b.
Also referred to as Etranacogene Dezaparvovec.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to learn whether CSL222 works, is safe, and is tolerated in this group.
- Who it's for
- Boys aged 12 to less than 18 years who have severe or moderately severe hemophilia B.
- What's involved
- Participants receive a single dose of CSL222 and then have regular check‑ups and lab tests to monitor clotting and safety.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 138 to 206, men only
You may be able to join if
- Key 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...
- 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.
You likely can't join if
- Key Exclusion Criteria for the Lead-in Period: History of FIX inhibitors or positive FIX inhibitor test at Screening (based on central laboratory...
- 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.
See the full eligibility criteria
- Key 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 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.
- Key Exclusion Criteria for the Lead-in Period: History of FIX inhibitors or positive FIX inhibitor test at Screening (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...
- 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).
- 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).
The study team makes the final eligibility decision.
Where it is enrolling
- Orange, California, United States
- San Diego, California, United States
- Gainesville, Florida, United States
- Atlanta, Georgia, United States
- Ann Arbor, Michigan, United States
- Memphis, Tennessee, United States
- Westmead, New South Wales, Australia
- Brisbane, Australia
- Vienna, Austria
- Leuven, Belgium
- Lille, France
- Marseille, France
- Ramat Gan, Israel
- Barcelona, Spain
- Madrid, Spain
- London, United Kingdom
- Oxford, United Kingdom
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, but 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, 138 months to 206 months. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Orange, California, United States; San Diego, California, United States; Gainesville, Florida, United States; Atlanta, Georgia, United States; Ann Arbor, Michigan, United States; Memphis, Tennessee, United States and 11 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.