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

NCT05962398 · tracked via the Priya Life Science UK 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

More CSL Behring trials in the UK

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 Recruiting

Single-arm Study of IgPro20 in Adults With Secondary Immune Deficiencies Due to Hematologic Malignancies Treated With B-cell Targeting Chimeric Antigen Receptor T-cell and T-cell Redirecting Therapies Recruiting

Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis Recruiting

Combined Dose-Finding and CV Outcomes Study With CSL300 (Clazakizumab) in Adult Subjects With ESKD Undergoing Dialysis (POSIBIL6ESKD) Recruiting

A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE) Recruiting

Other trials for the same condition

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 Recruiting

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B Recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B Active, not recruiting

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy Active, not recruiting

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 the UK