NCT06003387

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)

Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 35 (estimated)
• Sponsor: CSL Behring · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	CSL222 (AAV5-hFIXco-Padua)	Administered as a single IV infusion.

Timeline

• Start date: 2024-01-30
• Primary completion: 2028-10-04
• Completion: 2032-04-02
• First posted: 2023-08-22
• Last updated: 2026-03-17

Locations

26 sites across 12 countries: United States, Australia, Canada, Hong Kong, Israel, Mexico, Saudi Arabia, Singapore, South Africa, South Korea, Taiwan, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study