Clinical trial NCT05962398: Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
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
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Hemophilia B
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2023-07-27
Enrollment: 56
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult male participants with hemophilia B who were previously treated with CSL222 in study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).

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).


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

Primary outcomes:
measure: Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs)
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Percentage of Participants With SAEs and AESIs
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Number of SAEs and AESIs
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
Secondary outcomes:
measure: Annualized Bleeding Rate (ABR)
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Number of Participants With Zero Total, Spontaneous, Joint, and Traumatic Bleeding Episodes
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Endogenous Factor IX (FIX) Activity
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Change from Baseline by Visit in Endogenous FIX Activity
timeFrame: At Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Annualized Consumption of FIX Replacement Therapy
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Annualized Infusion Rate of FIX Replacement Therapy
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Number of Participants Remaining Free of Continuous FIX Prophylaxis
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Percentage of Participants Remaining Free of Continuous FIX Prophylaxis
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Number of New Target Joints
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Percentage Resolution of Pre-existing Target Joints
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) Index Value
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Change From Baseline in EQ-5D-5L Index Value
timeFrame: Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
measure: EQ-5D VAS Score
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Change From Baseline in EQ-5D VAS Score
timeFrame: Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Hemophilia Quality of Life Questionnaire (Hem-A-QoL) Total Score
timeFrame: From Year 5 after administration of CSL222 in parent study up to Year 15
measure: Change From Baseline in Hem-A-QoL Total Score
timeFrame: Baseline (in parent study), From Year 5 after administration of CSL222 in parent study up to Year 15
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: 10-15 Phoenix Childrens Hospital
state: Arizona
city: Little Rock
country: United States
facility: 10-14 Arkansas Children's Hospital - Pharmacology
state: Arkansas
city: Los Angeles
country: United States
facility: 10-22 Orthopaedic Institute for Children
state: California
city: Los Angeles
country: United States
facility: 10-21 Children's Hospital of Los Angeles
state: California
city: Sacramento
country: United States
facility: 10-63 UC Davis Medical Center
state: California
city: San Diego
country: United States
facility: 10-12 University of California, San Diego (UCSD)
state: California
city: Aurora
country: United States
facility: 10-25 University of Colorado Denver
state: Colorado
city: Tampa
country: United States
facility: 10-19 University of South Florida
state: Florida
city: Ann Arbor
country: United States
facility: 10-10 University of Michigan Medical Center
state: Michigan
city: Chapel Hill
country: United States
facility: 10-16 University of North Carolina at Chapel Hill
state: North Carolina
city: Portland
country: United States
facility: 10-18 Oregon Health & Science University
state: Oregon
city: Nashville
country: United States
facility: 10-20 Vanderbilt University Medical Center
state: Tennessee
city: Salt Lake City
country: United States
facility: 10-26 University of Utah
state: Utah
city: Seattle
country: United States
facility: 10-62 Washington Institute for Coagulation
state: Washington
city: Brussels
country: Belgium
facility: 11-31 Cliniques Universitaires Saint-Luc
city: Leuven
country: Belgium
facility: 11-30 Universitair Ziekenhuis Leuven
city: Copenhagen
country: Denmark
facility: 12-32 Rigshospitalet
city: Berlin
country: Germany
facility: 15-42 Vivantes Klinikum im Friedrichshain - Landsberger Allee
city: Dublin
country: Ireland
facility: 19-56 St James Hospital - Ireland
city: Dublin
country: Ireland
facility: 19-56 St. James Hospital
city: Amsterdam Zuidoost
country: Netherlands
facility: 13-33 Amsterdam Universitair Medische Centra (UMC)
city: Groningen
country: Netherlands
facility: 13-35 University Medical Center Groningen
city: Rotterdam
country: Netherlands
facility: 13-34 Erasmus University Medical Center
city: Utrecht
country: Netherlands
facility: 13-36 Universitair Medisch Centrum Utrecht
city: Malmo
country: Sweden
facility: 16-43 Skane University Hospital
city: London
country: United Kingdom
facility: 18-52 Barts Health NHS Trust
state: England
city: Southampton
country: United Kingdom
facility: 18-52, 18-54 University Hospital Southampton NHS Foundation Trust
city: Southampton
country: United Kingdom
facility: 18-53 University Hospital Southampton NHS Foundation Trust
Study officials:
affiliation: CSL Behring
name: Study Director
role: STUDY_DIRECTOR
References: