Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
IX-TEND 3003
An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B
2 other identifiers
observational
56
8 countries
28
Brief 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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Aug 2023
Longer than P75 for all trials
28 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 18, 2023
CompletedFirst Posted
Study publicly available on registry
July 27, 2023
CompletedStudy Start
First participant enrolled
August 30, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 23, 2035
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 23, 2035
July 18, 2025
July 1, 2025
11.6 years
July 18, 2023
July 17, 2025
Conditions
Outcome Measures
Primary Outcomes (3)
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
Secondary Outcomes (16)
Annualized Bleeding Rate (ABR)
From Year 5 after administration of CSL222 in parent study up to Year 15
Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes
From Year 5 after administration of CSL222 in parent study up to Year 15
Endogenous Factor IX (FIX) Activity
From Year 5 after administration of CSL222 in parent study up to Year 15
Change from Baseline by Visit in Endogenous FIX Activity
At Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
Annualized Consumption of FIX Replacement Therapy
From Year 5 after administration of CSL222 in parent study up to Year 15
- +11 more secondary outcomes
Study Arms (1)
CSL222
Participants who received CSL222 and completed either the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891) will be followed up from Year 5 after administration of CSL222 in parent study up to Year 15.
Interventions
Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).
Eligibility Criteria
Adult male participants with hemophilia B who were previously treated with CSL222 in study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).
You may qualify if:
- 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.
You may not qualify if:
- Not Applicable
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CSL Behringlead
Study Sites (28)
10-15 Phoenix Childrens Hospital
Phoenix, Arizona, 85016, United States
10-14 Arkansas Children's Hospital - Pharmacology
Little Rock, Arkansas, 72202, United States
10-22 Orthopaedic Institute for Children
Los Angeles, California, 90007, United States
10-21 Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
10-63 UC Davis Medical Center
Sacramento, California, 95817, United States
10-12 University of California, San Diego (UCSD)
San Diego, California, 92121, United States
10-25 University of Colorado Denver
Aurora, Colorado, 80045, United States
10-19 University of South Florida
Tampa, Florida, 33612, United States
10-10 University of Michigan Medical Center
Ann Arbor, Michigan, 48109, United States
10-16 University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599, United States
10-18 Oregon Health & Science University
Portland, Oregon, 97239, United States
10-20 Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
10-28 University of Texas Health Science Center
Houston, Texas, 77030, United States
10-26 University of Utah
Salt Lake City, Utah, 84108, United States
10-62 Washington Institute for Coagulation
Seattle, Washington, 98104, United States
11-31 Cliniques Universitaires Saint-Luc
Brussels, 1200, Belgium
11-30 Universitair Ziekenhuis Leuven
Leuven, 3000, Belgium
12-32 Rigshospitalet
Copenhagen, 2100, Denmark
15-42 Vivantes Klinikum im Friedrichshain - Landsberger Allee
Berlin, 10249, Germany
19-56 St. James Hospital
Dublin, D08 A978, Ireland
13-33 Amsterdam Universitair Medische Centra (UMC)
Amsterdam-Zuidoost, 1105 AZ, Netherlands
13-35 University Medical Center Groningen
Groningen, 9713 GZ, Netherlands
13-34 Erasmus University Medical Center
Rotterdam, Netherlands
13-36 Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
16-43 Skane University Hospital
Malmo, SE-205 02, Sweden
18-52 Barts Health NHS Trust
London, England, E1 1FR., United Kingdom
18-54 Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
18-53 University Hospital Southampton NHS Foundation Trust
Southampton, SO16 6YD, United Kingdom
Related Publications (1)
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key NS, Lattimore SU, Leebeek FWG, Miesbach WA, Recht M, Monahan PE, Le Quellec S, Pipe SW. Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years. Blood Adv. 2025 Jul 22;9(14):3543-3552. doi: 10.1182/bloodadvances.2024015291.
PMID: 40188458DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
CSL Behring
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 18, 2023
First Posted
July 27, 2023
Study Start
August 30, 2023
Primary Completion (Estimated)
March 23, 2035
Study Completion (Estimated)
March 23, 2035
Last Updated
July 18, 2025
Record last verified: 2025-07
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP
- Time Frame
- Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
- Access Criteria
- Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.