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Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up post-dose: at months 6,12, and 18
Awards & highlights
Study Summary
This trial will assess if a new drug is safe for people with hemophilia B and if it helps stop bleeding.
Who is the study for?
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.Check my eligibility
What is being tested?
The trial is testing the safety and effectiveness of CSL222 (Etranacogene Dezaparvovec), a gene therapy in adults with hemophilia B who already have AAV5 neutralizing antibodies. The goal is to see if this treatment can reduce the risk of bleeding by providing a functional version of the FIX gene.See study design
What are the potential side effects?
Potential side effects may include immune reactions due to pre-existing antibodies against AAV5 vector used in the gene therapy which could lead to reduced efficacy or allergic responses. Liver enzyme elevations are possible given that it's a common site for vector entry and transgene expression.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ post-dose: at months 6,12, and 18
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~post-dose: at months 6,12, and 18
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Annualized Bleeding Rate (ABR)
Secondary outcome measures
ABR for Spontaneous Bleeding Episodes, Joint Bleeding Episodes, and FIX-treated Bleeding Episodes Separately
Annualized Consumption of FIX Replacement Therapy
Annualized Infusion Rate of FIX Replacement Therapy
+29 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: CSL222Experimental Treatment1 Intervention
Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10^13 genome copies per kilogram (gc/kg) on Day D.
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Who is running the clinical trial?
CSL BehringLead Sponsor
194 Previous Clinical Trials
1,211,031 Total Patients Enrolled
18 Trials studying Hemophilia B
4,286 Patients Enrolled for Hemophilia B
Study DirectorStudy DirectorCSL Behring LLC
1,211 Previous Clinical Trials
489,237 Total Patients Enrolled
18 Trials studying Hemophilia B
922 Patients Enrolled for Hemophilia B
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Has CSL222 attained regulatory approval from the US Food and Drug Administration?
"Our researchers at Power have judged CSL222's safety to be a 3, as its Phase 3 trial has revealed positive efficacy results and numerous studies that validate the drug's security."
Answered by AI
Is enrollment for this clinical research open at the moment?
"As per clinicaltrials.gov, this research is not presently accepting participants; however, the trial was first posted on December 1st 2023 and its details were most recently updated on September 26th 2023. At this moment in time, there are 98 other studies actively recruiting patients."
Answered by AI
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