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 (26) 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:
• 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)
RECRUITING
San Diego
Michigan
University of Michigan
RECRUITING
Ann Arbor
Other Locations
Australia
Royal Adelaide Hospital
RECRUITING
Adelaide
Royal Prince Alfred Hospital
RECRUITING
Camperdown
Royal Brisbane Hospital
RECRUITING
Herston
The Alfred Hospital
RECRUITING
Melbourne
Canada
McMaster University - Hamilton
RECRUITING
Hamilton
Hong Kong Special Administrative Region
Queen Mary Hospital
RECRUITING
Hong Kong
Prince of Wales Hospital Chinese University of Hong Kong
RECRUITING
Shatin
Israel
Sheba Medical Center
RECRUITING
Tel Litwinsky
Mexico
Centro de Investigacion Clinica GRAMEL S.C.
RECRUITING
Mexico City
Republic of Korea
Kyungpook National University Hospital
RECRUITING
Daegu
Kyung Hee University Hospital at Gangdong
RECRUITING
Seoul
Severance Hospital, Yonsei University Health System
RECRUITING
Seoul
Saudi Arabia
King Faisal Specialist Hospital and Research Center
RECRUITING
Riyadh
Singapore
National University Hospital
RECRUITING
Singapore
Singapore General Hospital
RECRUITING
Singapore
South Africa
Haemophilia Comprehensive Care Centre
RECRUITING
Johannesburg
Taiwan
Changhua Christian Hospital (CCH)
RECRUITING
Chang-hua
Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)
RECRUITING
Kaohsiung City
Taichung Veterans General Hospital -
RECRUITING
Taichung
National Taiwan University Hospital
RECRUITING
Taipei
Tri-Service General Hospital
RECRUITING
Taipei
Turkey
Ege University Medical Faculty
RECRUITING
Bornova
Gaziantep University Sahinbey Research and Practice Hospital
RECRUITING
Gaziantep
Özel Acibadem Adana Hastanesi
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.