Enrolling by invitation NCT05962398
Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AAV5-hFIXco-Padua.
- Who it may be relevant to
- Registry conditions: Hemophilia B. Basic parameters: from 18 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Belgium, Denmark, Germany, Ireland +3
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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).
Interventions
- Genetic AAV5-hFIXco-Padua
Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).
Primary outcome measures
- Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Percentage of Participants With SAEs and AESIs [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Number of SAEs and AESIs [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
Secondary outcome measures (12)
- Annualized Bleeding Rate (ABR) [Time frame: 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 [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Endogenous Factor IX (FIX) Activity [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Change from Baseline by Visit in Endogenous FIX Activity [Time frame: 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 [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Annualized Infusion Rate of FIX Replacement Therapy [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Number of Participants Remaining Free of Continuous FIX Prophylaxis [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Percentage of Participants Remaining Free of Continuous FIX Prophylaxis [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Number of New Target Joints [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Percentage Resolution of Pre-existing Target Joints [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value [Time frame: From Year 5 after administration of CSL222 in parent study up to Year 15]
- Change From Baseline in EQ-5D-5L Index Value [Time frame: Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 15 centers
- 10-15 Phoenix Childrens Hospital — Phoenix
- 10-14 Arkansas Children's Hospital - Pharmacology — Little Rock
- 10-22 Orthopaedic Institute for Children — Los Angeles
- 10-21 Children's Hospital of Los Angeles — Los Angeles
- 10-63 UC Davis Medical Center — Sacramento
- 10-12 University of California, San Diego (UCSD) — San Diego
- 10-25 University of Colorado Denver — Aurora
- 10-19 University of South Florida — Tampa
- … and 7 more centers
Netherlands · 4 centers
- 13-33 Amsterdam Universitair Medische Centra (UMC) — Amsterdam-Zuidoost
- 13-35 University Medical Center Groningen — Groningen
- 13-34 Erasmus University Medical Center — Rotterdam
- 13-36 Universitair Medisch Centrum Utrecht — Utrecht
United Kingdom · 3 centers
- 18-52 Barts Health NHS Trust — London
- 18-54 Addenbrooke's Hospital — Cambridge
- 18-53 University Hospital Southampton NHS Foundation Trust — Southampton
Belgium · 2 centers
- 11-31 Cliniques Universitaires Saint-Luc — Brussels
- 11-30 Universitair Ziekenhuis Leuven — Leuven
Denmark · 1 center
- 12-32 Rigshospitalet — Copenhagen
Germany · 1 center
- 15-42 Vivantes Klinikum im Friedrichshain - Landsberger Allee — Berlin
Ireland · 1 center
- 19-56 St. James Hospital — Dublin
Sweden · 1 center
- 16-43 Skane University Hospital — Malmö
Publications
- 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 40188458
Identifiers
NCT: NCT05962398 · CSL222_3003 · EU CT Number