NCT07080905

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: CSL Behring · Industry
• Sex: Male
• Age: 138 Months – 206 Months
• Healthy volunteers: Not accepted

Summary

This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua)	Administered as a single IV infusion.

Timeline

• Start date: 2025-07-28
• Primary completion: 2033-10-24
• Completion: 2033-10-24
• First posted: 2025-07-23
• Last updated: 2026-04-16

Locations

10 sites across 5 countries: United States, Austria, Belgium, Israel, United Kingdom

Regulatory

• FDA-regulated drug study
• FDA-regulated device study