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Gene Therapy

CSL222 Gene Therapy for Hemophilia B

Phase 3
Recruiting
Research Sponsored by CSL Behring
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: months 6 to 18
Awards & highlights
No Placebo-Only Group
Pivotal Trial

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.
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.
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 weeks
Treatment ~ Varies
Follow Up ~post-dose: months 6 to 18
This trial's timeline: 3 weeks for screening, Varies for treatment, and post-dose: months 6 to 18 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Annualized Bleeding Rate (ABR)
Secondary study objectives
Change From Baseline in the EQ-5D-5L Index Scores
Change From Baseline in the Hem-A-QoL Total Score and Treatment Domain Score
Change from Baseline in the EQ-5D-5L VAS Score
+4 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial 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.

Find a Location

Who is running the clinical trial?

CSL BehringLead Sponsor
198 Previous Clinical Trials
1,204,786 Total Patients Enrolled
19 Trials studying Hemophilia B
4,309 Patients Enrolled for Hemophilia B
Study DirectorStudy DirectorCSL Behring LLC
1,275 Previous Clinical Trials
499,150 Total Patients Enrolled
18 Trials studying Hemophilia B
926 Patients Enrolled for Hemophilia B
~23 spots leftby Oct 2028
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