35 Participants Needed

CSL222 Gene Therapy for Hemophilia B

Recruiting at 17 trial locations
TR
Overseen ByTrial Registration Coordinator
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: CSL Behring
Must be taking: FIX prophylaxis
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

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?

Research shows that etranacogene dezaparvovec, a gene therapy for Hemophilia B, provides sustained production of factor IX (a protein needed for blood clotting) for more than 2 years, reducing the need for regular treatments and preventing bleeding in most patients.12345

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

SD

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

I have severe or moderately severe hemophilia B and am on regular treatment.
Acceptance to barrier contraception protection for 1 year starting the day of CSL222 treatment
I have been treated with FIX therapy for more than 150 days.
See 5 more

Exclusion Criteria

I have had inhibitors to Factor IX or tested positive for them.
ALT > 2 × the upper limit of normal (ULN) at Screening and Visit L-Final (based on central laboratory results)
I have a condition that makes me bleed more easily, but it's not hemophilia B.
See 7 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous (IV) infusion of CSL222 gene therapy

1 day
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

18 months
Regular visits (frequency not specified)

Treatment Details

Interventions

  • CSL222
Trial Overview 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.
Participant Groups
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 1.

CSL222 is already approved in European Union, United States for the following indications:

🇪🇺
Approved in European Union as HEMGENIX for:
  • Hemophilia B
🇺🇸
Approved in United States as HEMGENIX for:
  • Hemophilia B

Find a Clinic Near You

Who Is Running the Clinical Trial?

CSL Behring

Lead Sponsor

Trials
204
Recruited
1,207,000+
Dr. Paul McKenzie profile image

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 profile image

Dr. Bill Mezzanotte

CSL Behring

Chief Medical Officer since 2021

MD from Duke University

Findings from Research

Etranacogene dezaparvovec is the first approved gene therapy for adults with severe and moderately severe hemophilia B, allowing most patients to achieve stable factor IX levels after a single infusion, which significantly reduces the need for regular treatment and associated bleeding risks.
The therapy has shown efficacy even in patients with preexisting neutralizing antibodies and has a relatively low risk of transaminitis, but long-term data is needed to confirm the durability of factor IX levels and overall safety.
Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B.Castaman, G., Coppens, M., Pipe, SW.[2023]
Etranacogene dezaparvovec (Hemgenix®) is a gene therapy approved in the USA for treating adults with hemophilia B, specifically for those on FIX prophylaxis or with serious bleeding episodes.
The therapy received a positive opinion in the EU in December 2022, marking significant progress in its development and approval for treating congenital factor IX deficiency.
Etranacogene Dezaparvovec: First Approval.Heo, YA.[2023]
Valoctocogene roxaparvovec (Roctavian) and etranacogene dezaparavovec (Hemgenix) are effective gene therapies for hemophilia A and B, respectively, with phase 3 studies showing significant increases in clotting factor levels and reductions in bleeding events among participants (n=134 for Roctavian and n=54 for Hemgenix).
Both therapies were associated with manageable safety profiles, primarily involving elevated liver function tests, and they improved patients' quality of life by mimicking natural clotting factor production, offering a promising alternative to traditional replacement therapies.
Valoctocogene Roxaparvovec and Etranacogene Dezaparavovec: Novel Gene Therapies for Hemophilia A and B.Dougherty, JA., Dougherty, KM.[2023]

References

Etranacogene dezaparvovec for the treatment of adult patients with severe and moderately severe hemophilia B. [2023]
Etranacogene Dezaparvovec: First Approval. [2023]
Valoctocogene Roxaparvovec and Etranacogene Dezaparavovec: Novel Gene Therapies for Hemophilia A and B. [2023]
Etranacogene dezaparvovec for hemophilia B gene therapy. [2023]
Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B. [2023]
Etranacogene dezaparvovec-drlb gene therapy for patients with hemophilia B (congenital factor IX deficiency). [2023]
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