CSL222 Gene Therapy for Hemophilia B
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, you must have been on stable FIX prophylaxis for at least 2 months before screening.
What data supports the effectiveness of the treatment CSL222 for Hemophilia B?
What makes the treatment CSL222 unique for hemophilia B?
CSL222, also known as Etranacogene dezaparvovec, is unique because it is the first gene therapy approved for hemophilia B, offering a potential long-term solution by using a virus to deliver a healthy version of the gene responsible for producing factor IX, which is missing or defective in patients with this condition.12356
What is the purpose of this trial?
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
Research Team
Study Director
Principal Investigator
CSL Behring
Eligibility Criteria
Adults with severe or moderately severe hemophilia B who have been on stable clotting factor IX (FIX) prophylaxis, and have detectable AAV5 neutralizing antibodies. Participants must have had over 150 days of prior FIX therapy and be able to use eDiaries correctly. They should agree to use barrier contraception for a year post-treatment and not have any history of FIX inhibitors, severe liver impairment, uncontrolled infections like HIV or hepatitis C, thrombocytopenia, allergies to steroids or CSL222 components, previous gene therapy treatments, or other conditions causing increased bleeding.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intravenous (IV) infusion of CSL222 gene therapy
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- CSL222
CSL222 is already approved in European Union, United States for the following indications:
- Hemophilia B
- Hemophilia B
Find a Clinic Near You
Who Is Running the Clinical Trial?
CSL Behring
Lead Sponsor
Dr. Paul McKenzie
CSL Behring
Chief Executive Officer since 2023
PhD in Chemical Engineering from Carnegie Mellon University, B.S. in Chemical Engineering from the University of Pennsylvania
Dr. Bill Mezzanotte
CSL Behring
Chief Medical Officer since 2021
MD from Duke University