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Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults with Hemophilia B with Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (NAbs)
EU CTIS ID: 2023-509590-23-00
What this study is testing
The primary objective of this study is to assess whether there is a clinically significant correlation of pretreatment AAV5 NAb titers on the risk of bleeding during the 52 weeks following CSL222 treatment after the establishment of stable FIX expression (Months 7 to 18 postdose) compared to standard of care continuous routine FIX prophylaxis during the ≥ 6-month Lead-in Period.
- 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) Age ≥ 18 years and considered legally an adult, as defined by country regulations.
- 2) Has congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to [≤] 2% of normal circulating FIX) for which the subject is on continuous routine FIX prophylaxis.
- 3) Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results).
- 4) Has greater than (>) 150 previous exposure days to FIX replacement therapy
- 5) Has been on stable FIX prophylaxis for at least 2 months before Screening.
- 6) Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator.
You likely can't join if
- 1) History of FIX inhibitors or positive FIX inhibitor test at Prescreening, Screening or Visit L-Final (based on central laboratory results).
- 10) Receipt of an experimental agent or device within 60 days before Screening until the end of the study.
- 2) Screening or Visit L-Final laboratory values (based on central laboratory results) of total bilirubin > 2 × the upper limit of normal (ULN) (except if caused by Gilbert’s syndrome).
- 3) Screening or Visit L-Final laboratory values (based on central laboratory results).of any of the following laboratory abnormalities: • Alanine aminotransferase (ALT) > 2 × the ULN • Aspartate aminotransferase (AST) > 2 × the ULN • Alkaline phosphatase > 2 × the ULN • Serum creatinine > 2 × the ULN • Hemoglobin < 8 g/dL
- 4) Any condition other than hemophilia B resulting in an increased bleeding tendency.
- 5) Thrombocytopenia, defined as a platelet count 50 × 10^9/L, at Screening or Visit L-Final (based on central laboratory results).
See the full eligibility criteria
- 1) Age ≥ 18 years and considered legally an adult, as defined by country regulations.
- 2) Has congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to [≤] 2% of normal circulating FIX) for which the subject is on continuous routine FIX prophylaxis.
- 3) Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results).
- 4) Has greater than (>) 150 previous exposure days to FIX replacement therapy
- 5) Has been on stable FIX prophylaxis for at least 2 months before Screening.
- 6) Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator.
- 7) Acceptance to adhere to contraception
- 8) Able to provide informed consent after receipt of verbal and written information about the study.
- 9) Investigator believes that the participant (or the participant’s legally acceptable representative[s]) understands the nature, scope, and possible consequences of the study and is able to adhere to the study procedures.
- 1) History of FIX inhibitors or positive FIX inhibitor test at Prescreening, Screening or Visit L-Final (based on central laboratory results).
- 10) Receipt of an experimental agent or device within 60 days before Screening until the end of the study.
- 2) Screening or Visit L-Final laboratory values (based on central laboratory results) of total bilirubin > 2 × the upper limit of normal (ULN) (except if caused by Gilbert’s syndrome).
- 3) Screening or Visit L-Final laboratory values (based on central laboratory results).of any of the following laboratory abnormalities: • Alanine aminotransferase (ALT) > 2 × the ULN • Aspartate aminotransferase (AST) > 2 × the ULN • Alkaline phosphatase > 2 × the ULN • Serum creatinine > 2 × the ULN • Hemoglobin < 8 g/dL
- 4) Any condition other than hemophilia B resulting in an increased bleeding tendency.
- 5) Thrombocytopenia, defined as a platelet count 50 × 10^9/L, at Screening or Visit L-Final (based on central laboratory results).
- 6) Any uncontrolled or untreated infection (human immunodeficiency virus [HIV], hepatitis B virus [HBV] and hepatitis C virus [HCV], or any other significant concurrent, uncontrolled medical condition 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.
- 7) Known history of allergy to corticosteroids or known medical condition that would require chronic administration of oral corticosteroids.
- 8) Known uncontrolled allergic conditions or allergy / hypersensitivity to any component of the CSL222 excipients (ie, sucrose, potassium chloride, potassium dihydrogen phosphate, sodium chloride, and disodium hydrogen phosphate).
- 9) Previous gene therapy treatment.
The study team makes the final eligibility decision.
Where it's taking place
- Mexico
- Brazil
- Singapore
- United States
- Hong Kong
- Turkey
- Canada
- Israel
- Taiwan
- United Kingdom
- South Africa
- Saudi Arabia
- Australia
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 all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Mexico; Brazil; Singapore; United States; Hong Kong; Turkey and 7 more location(s). 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.