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 Recruiting 20 enrolled CSL Behring

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2025-07-28
Primary completion: 2033-10-24
Completion: 2033-10-24

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CSL222	Gene therapy (AAV / viral vector)	2e+13 vg/kg	Intravenous

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

Summary

Timeline

Drugs

Indications