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Clinical Trials in the USA / NCT05962398
Enrolling by invitation Observational

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

NCT05962398 · tracked via the Priya Life Science USA 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
No
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)

FacilityCityRegionStatus
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 USA

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) Recruiting
Two Dose Levels of Privigen in Pediatric CIDP Recruiting
Combined Dose-Finding and CV Outcomes Study With CSL300 (Clazakizumab) in Adult Subjects With ESKD Undergoing Dialysis (POSIBIL6ESKD) Recruiting
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD) Recruiting
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B Recruiting

Other trials for the same condition

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
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) 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 Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency) Recruiting
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors 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 USA