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)
NCT ID: NCT06003387
Last Updated: 2025-12-12
Study Results
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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RECRUITING
PHASE3
35 participants
INTERVENTIONAL
2024-01-30
2032-04-02
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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CSL222
Participants will receive CSL222 as a single intravenous (IV) infusion of 2 × 10\^13 genome copies per kilogram (gc/kg) on Day 1.
CSL222 (AAV5-hFIXco-Padua)
Administered as a single IV infusion.
Interventions
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CSL222 (AAV5-hFIXco-Padua)
Administered as a single IV infusion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* •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.
Exclusion Criteria
* • 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:
* a) ALT \> 2 × the ULN
* b) AST \> 2 × the ULN
* c) Alkaline phosphatase \> 2 × the ULN
* d) Serum creatinine \> 2 × the ULN
* e) 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.
18 Years
ALL
No
Sponsors
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CSL Behring
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
CSL Behring
Locations
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University of California, San Diego (UCSD) - 84000460
San Diego, California, United States
University of Michigan - 84000285
Ann Arbor, Michigan, United States
Royal Prince Alfred Hospital - 03600044
Camperdown, New South Wales, Australia
Royal Brisbane Hospital - 03600045
Herston, Queensland, Australia
Royal Adelaide Hospital
Adelaide, South Australia, Australia
The Alfred Hospital - 03600043
Melbourne, Victoria, Australia
McMaster University - Hamilton - 12400017
Hamilton, Ontario, Canada
Queen Mary Hospital - 34400001
Hong Kong, , Hong Kong
Prince of Wales Hospital Chinese University of Hong Kong - 34400002
Shatin, , Hong Kong
Sheba Medical Center - 37600004
Tel Litwinsky, , Israel
Centro de Investigacion Clinica GRAMEL S.C. - 48400003
Mexico City, Mexico City, Mexico
King Faisal Specialist Hospital and Research Center - 68200001
Riyadh, , Saudi Arabia
National University Hospital - 70200002
Singapore, , Singapore
Haemophilia Comprehensive Care Centre - 71000001
Johannesburg, , South Africa
Kyungpook National University Hospital - 41000014
Daegu, , South Korea
Kyung Hee University Hospital at Gangdong - 41000015
Seoul, , South Korea
Severance Hospital, Yonsei University Health System - 41000013
Seoul, , South Korea
Tri-Service General Hospital - 15800012
Taipei, Neihu District, Taiwan
Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH) - 15800011
Kaohsiung City, Sanmin District, Taiwan
Changhua Christian Hospital (CCH) - 15800009
Chang-hua, , Taiwan
Taichung Veterans General Hospital - 15800007
Taichung, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Ege University Medical Faculty - 79200002
Bornova, , Turkey (Türkiye)
Gaziantep University Sahinbey Research and Practice Hospital - 79200004
Gaziantep, , Turkey (Türkiye)
Özel Acibadem Adana Hastanesi - 79200003
Seyhan, , Turkey (Türkiye)
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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2023-509590-23-00
Identifier Type: CTIS
Identifier Source: secondary_id
CSL222_3005
Identifier Type: -
Identifier Source: org_study_id
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