Actively Recruiting
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
Led by CSL Behring · Updated on 2026-03-17
35
Participants Needed
26
Research Sites
426 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Considered legally an adult according to country regulations
- Diagnosed with congenital severe or moderately severe hemophilia B with Factor IX deficiency of 2% or less of normal
- Has two consecutive detectable AAV5 neutralizing antibody titers before treatment
- Has had more than 150 previous exposure days to Factor IX replacement therapy
- Has been on stable Factor IX prophylaxis for at least 2 months before screening
- Can independently and accurately complete the electronic diary during the lead-in period
- Agrees to follow contraception guidelines
- Able to provide informed consent and understand the study procedures
You will not qualify if you...
- History or presence of Factor IX inhibitors at screening or prior to treatment
- Elevated total bilirubin levels over twice the upper limit of normal, except Gilbert's syndrome
- Elevated liver enzymes (ALT, AST), alkaline phosphatase, or serum creatinine over twice the upper limit of normal
- Hemoglobin level less than 8 g/dL
- Any bleeding disorder other than hemophilia B
- Low platelet count below 50 x 10^9/L at screening
- Any uncontrolled or untreated infections including HIV, hepatitis B or C
- Significant uncontrolled medical conditions affecting various organs or systems
- Known allergy to corticosteroids or need for chronic oral corticosteroid use
- Known uncontrolled allergic conditions or allergy to any CSL222 excipients
- Previous treatment with AAV5 gene therapy
- Use of experimental agents or devices within 60 days before screening until study end
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 26 locations
1
University of California, San Diego (UCSD)
San Diego, California, United States, 92121
Actively Recruiting
2
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
3
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia, 2050
Actively Recruiting
4
Royal Brisbane Hospital
Herston, Queensland, Australia, 4029
Actively Recruiting
5
Royal Adelaide Hospital
Adelaide, South Australia, Australia, 5000
Actively Recruiting
6
The Alfred Hospital
Melbourne, Victoria, Australia, 3004
Actively Recruiting
7
McMaster University - Hamilton
Hamilton, Ontario, Canada, L8N 3Z5
Actively Recruiting
8
Queen Mary Hospital
Hong Kong, Hong Kong, 999077
Actively Recruiting
9
Prince of Wales Hospital Chinese University of Hong Kong
Shatin, Hong Kong, 999077
Actively Recruiting
10
Sheba Medical Center
Tel Litwinsky, Israel, 5265601
Actively Recruiting
11
Centro de Investigacion Clinica GRAMEL S.C.
Mexico City, Mexico City, Mexico, 3720
Actively Recruiting
12
King Faisal Specialist Hospital and Research Center
Riyadh, Saudi Arabia, 11471
Actively Recruiting
13
National University Hospital
Singapore, Singapore, 110974
Actively Recruiting
14
Singapore General Hospital
Singapore, Singapore, 169608
Actively Recruiting
15
Haemophilia Comprehensive Care Centre
Johannesburg, South Africa, 2193
Actively Recruiting
16
Kyungpook National University Hospital
Daegu, South Korea, 41944
Actively Recruiting
17
Kyung Hee University Hospital at Gangdong
Seoul, South Korea, 05278
Actively Recruiting
18
Severance Hospital, Yonsei University Health System
Seoul, South Korea, 3722
Actively Recruiting
19
Tri-Service General Hospital
Taipei, Neihu District, Taiwan, 114
Actively Recruiting
20
Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)
Kaohsiung City, Sanmin District, Taiwan, 80756
Actively Recruiting
21
Changhua Christian Hospital (CCH)
Chang-hua, Taiwan, 500
Actively Recruiting
22
Taichung Veterans General Hospital -
Taichung, Taiwan, 40705
Actively Recruiting
23
National Taiwan University Hospital
Taipei, Taiwan, 100225
Actively Recruiting
24
Ege University Medical Faculty
Bornova, Turkey (Türkiye), 35100
Actively Recruiting
25
Gaziantep University Sahinbey Research and Practice Hospital
Gaziantep, Turkey (Türkiye), 27310
Actively Recruiting
26
Özel Acibadem Adana Hastanesi
Seyhan, Turkey (Türkiye), 01130
Actively Recruiting
Research Team
T
Trial Registration Coordinator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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