Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies
Status: Recruiting
Location: See all (23) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• Age \>= 18 years and considered legally an adult, as defined by country regulations.
• Has congenital hemophilia B with known severe or moderately severe FIX deficiency (less than or equal to \[\<=\] 2% of normal circulating FIX) for which the participant is on continuous routine FIX prophylaxis.
• Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results).
• Has greater than (\>) 150 previous exposure days to FIX replacement therapy.
• Has been on stable FIX prophylaxis for at least 2 months before Screening.
• Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator.
• Acceptance to adhere to contraception guidelines.
• Able to provide informed consent after receipt of verbal and written information about the study.
• Investigator believes that the participant (or the participant's legally acceptable representative\[s\]) understands the nature, scope, and possible consequences of the study and is able to adhere to the study procedures.
Locations
United States
California
University of California, San Diego (UCSD) - 84000460
RECRUITING
San Diego
Michigan
University of Michigan - 84000285
RECRUITING
Ann Arbor
Other Locations
Australia
Royal Prince Alfred Hospital - 03600044
RECRUITING
Camperdown
Royal Brisbane Hospital - 03600045
RECRUITING
Herston
The Alfred Hospital - 03600043
RECRUITING
Melbourne
Canada
McMaster University - Hamilton - 12400017
RECRUITING
Hamilton
Hong Kong Special Administrative Region
Queen Mary Hospital - 34400001
RECRUITING
Hong Kong
Prince of Wales Hospital Chinese University of Hong Kong - 34400002
RECRUITING
Shatin
Israel
Sheba Medical Center - 37600004
RECRUITING
Tel Litwinsky
Mexico
Centro de Investigacion Clinica GRAMEL S.C. - 48400003
RECRUITING
Mexico City
Republic of Korea
Kyungpook National University Hospital - 41000014
RECRUITING
Daegu
Kyung Hee University Hospital at Gangdong - 41000015
RECRUITING
Seoul
Severance Hospital, Yonsei University Health System - 41000013
RECRUITING
Seoul
Saudi Arabia
King Faisal Specialist Hospital and Research Center - 68200001
RECRUITING
Riyadh
Singapore
National University Hospital - 70200002
RECRUITING
Singapore
South Africa
Haemophilia Comprehensive Care Centre - 71000001
RECRUITING
Johannesburg
Taiwan
Changhua Christian Hospital (CCH) - 15800009
RECRUITING
Chang-hua
Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH) - 15800011
RECRUITING
Kaohsiung City
Taichung Veterans General Hospital - 15800007
RECRUITING
Taichung
Tri-Service General Hospital - 15800012
RECRUITING
Taipei
Turkey
Ege University Medical Faculty - 79200002
RECRUITING
Bornova
Gaziantep University Sahinbey Research and Practice Hospital - 79200004
RECRUITING
Gaziantep
Özel Acibadem Adana Hastanesi - 79200003
RECRUITING
Seyhan
Contact Information
Primary
Trial Registration Coordinator
clinicaltrials@cslbehring.com
1-610-878-4697
Time Frame
Start Date:2024-01-30
Estimated Completion Date:2032-04-02
Participants
Target number of participants:35
Treatments
Experimental: CSL222
Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10\^13 genome copies per kilogram (gc/kg) on Day 1.