Long-term Follow-up of Adult Men Treated for Hemophilia B With Gene Therapy
Follows adult men with hemophilia B who previously received etranacogene dezaparvovec gene therapy.
Key facts
- Who runs it
- CSL Behring
- Where
- Leuven and Sint-Lambrechts-Woluwe, BelgiumSee the 2 sitesAlso in 7 other countries, 26 sites
- Trial code
- Registry title
- Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
- Status
- Enrolling by invitation
- Phase
- Phase 2/3
- People taking part
- 56 people, planned
- Started
- 30 Aug 2023
- Expected to end
- 23 Mar 2035
- Type of study
- Observational
- Treatments tested
- AAV5-hFIXco-Padua
- Record updated
- 18 July 2025
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for men with hemophilia B who already received CSL222 in an earlier study. It follows them over the long term to check safety and effect.
- Age
- 18 years and older
- Sex
- Men only
- Earlier treatment
- Must have received CSL222 in study CSL222_2001 or CSL222_3001. Must have finished that study, or at least 5 years have passed since receiving CSL222.
The registry criteria, word for word
Show all 2 criteriaShow less
Who can take part
- 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
Who cannot take part
- Not Applicable
Where in Belgium
Also in 7 other countries, 26 sites
- UZ LeuvenLeuven, BelgiumNo longer taking part
- Cliniques Universitaires Saint-LucSint-Lambrechts-Woluwe, BelgiumNo longer taking part
About the trial
What the trial wants to find out
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).
The group in the trial
- 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.
Treatment: AAV5-hFIXco-Padua
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official title
- An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B
- Plain-language title
- Long-term Follow-up of Adult Men Treated for Hemophilia B With Gene TherapyGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Record updated
- 18 July 2025