Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

NCT05962398 · tracked via the Priya Life Science Ireland tracker

Sponsor

CSL Behring

Phase

Observational

Started

2023-08-30

Last updated

2025-07-18

Condition(s) studied

Hemophilia B

Investigational drug(s) / intervention(s)

AAV5-hFIXco-Padua

AAV5-hFIXco-Padua: Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).

Study summary

The primary purpose of this study is to assess the long-term safety and efficacy in male adults with hemophilia B who were treated with CSL222 (CSL222) in parent studies CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).

Eligibility

Sex

MALE

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: Received treatment with CSL222 in Study CSL222\_2001 or Study CSL222\_3001, and completed participation in Study CSL222\_2001 or Study CSL222\_3001 or at least 5 years have passed since receiving CSL222. Exclusion Criteria: Not Applicable

Primary outcome measure(s)

Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) — From Year 5 after administration of CSL222 in parent study up to Year 15
Percentage of Participants With SAEs and AESIs — From Year 5 after administration of CSL222 in parent study up to Year 15
Number of SAEs and AESIs — From Year 5 after administration of CSL222 in parent study up to Year 15

Trial sites (28)

Facility	City	Region
10-15 Phoenix Childrens Hospital	Phoenix	Arizona
10-14 Arkansas Children's Hospital - Pharmacology	Little Rock	Arkansas
10-22 Orthopaedic Institute for Children	Los Angeles	California
10-21 Children's Hospital of Los Angeles	Los Angeles	California
10-63 UC Davis Medical Center	Sacramento	California
10-12 University of California, San Diego (UCSD)	San Diego	California
10-25 University of Colorado Denver	Aurora	Colorado
10-19 University of South Florida	Tampa	Florida
10-10 University of Michigan Medical Center	Ann Arbor	Michigan
10-16 University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
10-18 Oregon Health & Science University	Portland	Oregon
10-20 Vanderbilt University Medical Center	Nashville	Tennessee
10-28 University of Texas Health Science Center	Houston	Texas
10-26 University of Utah	Salt Lake City	Utah
10-62 Washington Institute for Coagulation	Seattle	Washington
11-31 Cliniques Universitaires Saint-Luc	Brussels	Belgium
11-30 Universitair Ziekenhuis Leuven	Leuven	Belgium
12-32 Rigshospitalet	Copenhagen	Denmark
15-42 Vivantes Klinikum im Friedrichshain - Landsberger Allee	Berlin	Germany
19-56 St. James Hospital	Dublin	Ireland
13-33 Amsterdam Universitair Medische Centra (UMC)	Amsterdam-Zuidoost	Netherlands
13-35 University Medical Center Groningen	Groningen	Netherlands
13-34 Erasmus University Medical Center	Rotterdam	Netherlands
13-36 Universitair Medisch Centrum Utrecht	Utrecht	Netherlands
16-43 Skane University Hospital	Malmö	Sweden
18-52 Barts Health NHS Trust	London	England
18-54 Addenbrooke's Hospital	Cambridge	United Kingdom
18-53 University Hospital Southampton NHS Foundation Trust	Southampton	United Kingdom

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05962398 on ClinicalTrials.gov ↗ ← All trials in Ireland