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.
Plain-language title generated from the registry record
Registry title: Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
Key facts
- Where
- Ann Arbor, Aurora, Chapel Hill, Houston, Little Rock, Los Angeles, Nashville, Phoenix, Portland, Sacramento, Salt Lake City, San Diego, Seattle, and Tampa, United StatesSee the 15 sites
- Phase
- Phase 2/3
Where in United States
- 10-10 University of Michigan Medical CenterAnn Arbor, United StatesNo longer taking part
- 10-25 University of Colorado DenverAurora, United StatesNo longer taking part
- 10-16 University of North Carolina at Chapel HillChapel Hill, United StatesNo longer taking part
- 10-28 University of Texas Health Science CenterHouston, United StatesNo longer taking part
- 10-14 Arkansas Children's Hospital - PharmacologyLittle Rock, United StatesNo longer taking part
- 10-21 Children's Hospital of Los AngelesLos Angeles, United StatesNo longer taking part
- 10-22 Orthopaedic Institute for ChildrenLos Angeles, United StatesNo longer taking part
- 10-20 Vanderbilt University Medical CenterNashville, United StatesNo longer taking part
- 10-15 Phoenix Childrens HospitalPhoenix, United StatesNo longer taking part
- 10-18 Oregon Health & Science UniversityPortland, United StatesNo longer taking part
- 10-63 UC Davis Medical CenterSacramento, United StatesNo longer taking part
- 10-26 University of UtahSalt Lake City, United StatesNo longer taking part
- 10-12 University of California, San Diego (UCSD)San Diego, United StatesNo longer taking part
- 10-62 Washington Institute for CoagulationSeattle, United StatesNo longer taking part
- 10-19 University of South FloridaTampa, United StatesNo longer taking part
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?
Trial details
- Who runs it
- CSL Behring
- Trial code
- NCT05962398EU 2023-503765-37-00
- Record updated
- 18 July 2025
- Original description
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).Read full original descriptionShow less
An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B
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).
Inclusion: 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: Not Applicable.