Hemophilia B
Conditions
Interventions
Single group:JWK004 Single intravenous infusion administration
Sponsors
West China Hospital, Sichuan University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: Subjects who meet all the following criteria are eligible for enrollment: (1) Male, age = 18 years old; (2) Suffering from type B hemophilia, the endogenous FIX activity level is = 2 IU/dL (= 2%), as recorded by a certified clinical laboratory during screening. If the screening result is greater than 2% due to insufficient elution of FIX protein products, the severity of hemophilia B can be confirmed by historical evidence from certified clinical laboratories with = 2% FIX coagulant activity (FIX: C); (3) Based on the historical data in the subject's records/medical history, having previously been exposed to any recombinant and/or plasma derived FIX products for = 100 days; (4) Acceptable laboratory values: hemoglobin = 90g/L, platelets = 100 × 10 ^ 9/L, AST, ALT, alkaline phosphatase (laboratory indicators during screening) = 2 × Upper limit of normal value (ULN), bilirubin = 3 × ULN, serum creatinine = 108umol/L; (5) Able to understand the purpose, content, and potential risks of the experiment, voluntarily participate and sign an informed consent form; (6) Agree to use reliable contraceptive measures until the carrier sequence of the semen sample is tested negative twice in a row.
Exclusion criteria
Exclusion criteria: Those who meet any of the following criteria are not eligible for enrollment: (1) Positive medical history of FIX inhibitor test; (2) Positive FIX inhibitor test during screening or previous clinical symptoms or signs of reduced response to FIX administration; (3) During screening, the titer of AAV5 neutralizing antibody was greater than 1:100. (4) When filtering, one of the following situations exists: a. Hepatitis B surface antigen (HBsAg) is positive, and the copy number of hepatitis B virus deoxyribonucleic acid (HBV-DNA) is>the upper limit of normal value (ULN); b. Hepatitis C virus antibody (HCV-Ab) is positive, and the copy number of hepatitis C virus ribonucleic acid (HCV-RNA) is>ULN; c. Receiving antiviral treatment for hepatitis B or C; d. The human immunodeficiency virus (HIV) test is positive and the CD4+T lymphocyte count is = 200/mm3; (5) Have a history of chronic infections or other chronic diseases that researchers believe pose an unacceptable risk; (6) Any coagulation disorders other than hemophilia B; (7) Plan to undergo surgery within 6 months after the administration of JWK004 injection; (8) Have experienced arterial or venous thrombosis events within the first 6 months of screening (such as acute myocardial infarction, cerebrovascular disease, and venous thrombosis); (9) Active severe infection or any other significant accompanying, uncontrolled medical condition, including but not limited to kidney, liver, blood, gastrointestinal, endocrine, lung, nervous system, brain or mental illness, alcoholism, drug dependence, or any psychological disorder assessed by the researcher that may interfere with adherence to experimental protocol procedures or tolerance to JWK004 injection; (10) Known important medical conditions, including diffuse intravascular coagulation, fibrinolysis, and liver fibrosis, and studies suggest that they may interfere with the interpretation of safety or efficacy data. (11) A history of allergic reactions or shock to FIX formulations is known. (12) Known to have uncontrolled allergies or allergic reactions to any components of JWK004 injection excipients. (13) Previously received gene therapy or participated in interventional clinical studies within the past 12 weeks; (14) The researcher believes that the subject is not suitable to participate in any concurrent clinically significant major diseases or other situations in the study; (15) Unable or unwilling to comply with the visit and study evaluation schedule described in the clinical protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| FIX activity level and FIX protein concentration; | — |
Secondary
| Measure | Time frame |
|---|---|
| Hemophilia Joint Health Score;Quality of life score;Disability score;Physical activity score;Hemophilia Health Status Score;Adverse Events; | — |
Countries
CHINA
Contacts
Public ContactXINGCHEN PENG
West China Hospital, Sichuan University
Outcome results
None listed