Last updated 7 days ago
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)
35 patients around the world
Available in Mexico
CSL Behring
1Research sites
35Patients around the world
This study is for people with
Hemophilia
Hemophilia b
Requirements for the patient
From 18 Years
All Gender
Medical requirements
- 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 (≤ 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 > 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 barrier contraception protection for 1 year starting the day of CSL222 treatment.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.
- 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 that meet the definition of Severe Hepatic Impairment per Common Terminology Criteria for Adverse Events (CTCAE) (based on central laboratory results).ALT > 2 × the ULN at Screening or Visit L-Final laboratory values (based on central laboratory results).Any condition other than hemophilia B resulting in an increased bleeding tendency.Thrombocytopenia, defined as a platelet count below 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 gene therapy treatment.Receipt of an experimental agent or device within 60 days before Screening until the end of the study.
Sites
Centro de Investigación Clínica GRAMEL
Recruiting
SponsorCSL Behring
Study typeInterventional
Conditions
Hemophilia b
Requirements
From 18 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT06003387
