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Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

NCT ID: NCT05962398

Last Updated: 2025-07-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

56 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-08-30

Study Completion Date

2035-03-23

Brief Summary

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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).

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Detailed Description

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Conditions

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Hemophilia B

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

AAV5-hFIXco-Padua

Intervention Type GENETIC

Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).

Interventions

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AAV5-hFIXco-Padua

Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222\_2001 (NCT03489291) or CSL222\_3001 (NCT03569891).

Intervention Type GENETIC

Other Intervention Names

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Etranacogene dezaparvovec

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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CSL Behring

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

CSL Behring

Locations

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10-15 Phoenix Childrens Hospital

Phoenix, Arizona, United States

Site Status

10-14 Arkansas Children's Hospital - Pharmacology

Little Rock, Arkansas, United States

Site Status

10-22 Orthopaedic Institute for Children

Los Angeles, California, United States

Site Status

10-21 Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status

10-63 UC Davis Medical Center

Sacramento, California, United States

Site Status

10-12 University of California, San Diego (UCSD)

San Diego, California, United States

Site Status

10-25 University of Colorado Denver

Aurora, Colorado, United States

Site Status

10-19 University of South Florida

Tampa, Florida, United States

Site Status

10-10 University of Michigan Medical Center

Ann Arbor, Michigan, United States

Site Status

10-16 University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

10-18 Oregon Health & Science University

Portland, Oregon, United States

Site Status

10-20 Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

10-28 University of Texas Health Science Center

Houston, Texas, United States

Site Status

10-26 University of Utah

Salt Lake City, Utah, United States

Site Status

10-62 Washington Institute for Coagulation

Seattle, Washington, United States

Site Status

11-31 Cliniques Universitaires Saint-Luc

Brussels, , Belgium

Site Status

11-30 Universitair Ziekenhuis Leuven

Leuven, , Belgium

Site Status

12-32 Rigshospitalet

Copenhagen, , Denmark

Site Status

15-42 Vivantes Klinikum im Friedrichshain - Landsberger Allee

Berlin, , Germany

Site Status

19-56 St. James Hospital

Dublin, , Ireland

Site Status

13-33 Amsterdam Universitair Medische Centra (UMC)

Amsterdam-Zuidoost, , Netherlands

Site Status

13-35 University Medical Center Groningen

Groningen, , Netherlands

Site Status

13-34 Erasmus University Medical Center

Rotterdam, , Netherlands

Site Status

13-36 Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status

16-43 Skane University Hospital

Malmo, , Sweden

Site Status

18-52 Barts Health NHS Trust

London, England, United Kingdom

Site Status

18-54 Addenbrooke's Hospital

Cambridge, , United Kingdom

Site Status

18-53 University Hospital Southampton NHS Foundation Trust

Southampton, , United Kingdom

Site Status

Countries

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United States Belgium Denmark Germany Ireland Netherlands Sweden United Kingdom

References

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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.

Reference Type DERIVED
PMID: 40188458 (View on PubMed)

Other Identifiers

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EU CT Number

Identifier Type: OTHER

Identifier Source: secondary_id

CSL222_3003

Identifier Type: -

Identifier Source: org_study_id

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