- * History of FIX inhibitors or positive FIX inhibitor test at Prescreening, Screening or Visit L-Final (based on central laboratory results).
- * Screening or Visit L-Final laboratory values (based on central laboratory results) of total bilirubin \> 2 × the upper limit of normal (ULN) (except if caused by Gilbert's syndrome).
- * Screening or Visit L-Final laboratory values (based on central laboratory results) of any of the following laboratory abnormalities:
- 1. ALT \> 2 × the ULN
- 2. AST \> 2 × the ULN
- 3. Alkaline phosphatase \> 2 × the ULN
- 4. Serum creatinine \> 2 × the ULN
- 5. Hemoglobin less than (\<) 8 g/dL
- * Any condition other than hemophilia B resulting in an increased bleeding tendency.
- * Thrombocytopenia, defined as a platelet count \<50 × 10\^9/L, at Screening or Visit L Final (based on central laboratory results).
- * Any uncontrolled or untreated infection (human immunodeficiency virus \[HIV\], hepatitis B virus \[HBV\] and hepatitis C virus \[HCV\], or any other significant concurrent, uncontrolled medical condition including, but not limited to, renal, hepatic, cardiovascular, hematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral or psychiatric disease, alcoholism, drug dependency, or any other psychological disorder evaluated by the investigator to interfere with adherence to the clinical study protocol procedures or with the degree of tolerance to CSL222.
- * Known history of allergy to corticosteroids or known medical condition that would require chronic administration of oral corticosteroids.
- * Known uncontrolled allergic conditions or allergy / hypersensitivity to any component of the CSL222 excipients (ie, sucrose, potassium chloride, potassium dihydrogen phosphate, sodium chloride, and disodium hydrogen phosphate).
- * Previous AAV5 gene therapy treatment.
- * Receipt of an experimental agent or device within 60 days before Screening until the end of the study.
Hemophilia B
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)
NCT06003387 | PHASE 3 | INTERVENTIONAL
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.
Trial Information
If interested, contact clinicaltrials@cslbehring.com or +1 610-878-4697 for more information
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University of California, San Diego (UCSD)
San Diego,California,United States,92121
University of Michigan
Ann Arbor,Michigan,United States,48109
Royal Prince Alfred Hospital
Camperdown,New South Wales,Australia,2050
Royal Brisbane Hospital
Herston,Queensland,Australia,4029
The Alfred Hospital
Melbourne,Victoria,Australia,3004
McMaster University Hamilton
Hamilton,Ontario,Canada,L8N3Z5
Queen Mary Hospital
Hong Kong,Ontario,Hong Kong,999077
Prince of Wales Hospital Chinese University of Hong Kong
Shatin,Ontario,Hong Kong,999077
Sheba Medical Center
Tel Litwinsky,Ontario,Israel,5265601
Centro de Investigacion Clinica GRAMEL S.C.
Mexico City,Mexico City,Mexico,3720
King Faisal Specialist Hospital and Research Center
Riyadh,Mexico City,Saudi Arabia,11471
National University Hospital
Singapore,Mexico City,Singapore,110974
Haemophilia Comprehensive Care Centre
Johannesburg,Mexico City,South Africa,2193
Kyungpook National University Hospital
Daegu,Mexico City,South Korea,41944
Kyung Hee University Hospital at Gangdong
Seoul,Mexico City,South Korea,05278
Severance Hospital, Yonsei University Health System
Seoul,Mexico City,South Korea,3722
TriService General Hospital
Taipei,Neihu District,Taiwan,114
Kaohsiung Medical University ChungHo Memorial Hospital (KMUH)
Kaohsiung City,Sanmin District,Taiwan,80756
Changhua Christian Hospital (CCH)
Chang-hua,Sanmin District,Taiwan,500
Taichung Veterans General Hospital
Taichung,Sanmin District,Taiwan,40705
Ege University Medical Faculty
Bornova,Sanmin District,Turkey (Türkiye),35100
Gaziantep University Sahinbey Research and Practice Hospital
Gaziantep,Sanmin District,Turkey (Türkiye),27310
zel Acibadem Adana Hastanesi
Seyhan,Sanmin District,Turkey (Türkiye),01130
Study Eligibility Criteria
Additional Studies
Additional studies can be found at ClinicalTrials.gov