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)

Part of paid clinical trials in San Diego, California.

Sponsor
CSL Behring
Study ID
NCT06003387
Phase
PHASE3
Status
Recruiting
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Conditions

  • Hemophilia B

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • CSL222 (AAV5-hFIXco-Padua) — GENETIC
    Administered as a single IV infusion.

Study Details

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.

Key Dates

Start date
Jan 30, 2024
Status verified
Mar 2026
Primary completion
Oct 4, 2028
Completion
Apr 2, 2032

Study Design

Enrollment
35 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CSL222
    Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10\^13 genome copies per kilogram (gc/kg) on Day 1.

Primary Outcome Measure

Annualized Bleeding Rate (ABR) [ Time Frame: Months 7 to 18 after CSL222 treatment ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
University of California, San Diego (UCSD)San DiegoCalifornia92121
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University of MichiganAnn ArborMichigan48109
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Find similar trials in San Diego, CA

By research site
University of California, San Diego (UCSD)· San Diego, CAUniversity of Michigan· Ann Arbor, MI

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