Hemophilia B
Conditions
Interventions
Single arm:A single-dose intravenous infusion of BBM-H901 injection
Sponsors
Tongji Medical college of Huazhong University of Science&Technology
Eligibility
Sex/Gender
Male
Age
12 Years to 18 Years
Inclusion criteria
Inclusion criteria: 1. The subjects and/or their legal guardians fully understand the research purpose, nature, methods, and potential adverse reactions, voluntarily act as subjects, and sign an informed consent form; 2. Age and gender requirements: 12 years old = Patient age2% during screening is due to insufficient elution of FIX protein products, can be fully eluted and retested once; 4. Exposure days (EDs) = 75 for treatment with any recombinant and plasma derived FIX protein products; 5. The titer of anti BBM-H901 capsid neutralizing antibody = acceptable laboratory test value, and the titer of capsid binding antibody = acceptable laboratory test value; 6. In the medical records of the subjects, there have been bleeding events and/or injections of FIX products (including recombinant and plasma sources) within the 12 weeks prior to screening; 7. No allergic reactions or history related to FIX or intravenous administration of human immunoglobulin; 8. Without FIX inhibitors, there is no previous medical history of FIX inhibitors after 75EDs of FIX products; There are no clinical signs or symptoms of reduced response after FIX product infusion; 9. Obtain acceptable laboratory test values during screening and before administration (Day-1): a) Hemoglobin = 11 g/dL or = upper limit of normal values for peers b) Platelets = 100 × 10 ^ 9/L c) AST, ALT = 1.5 times the upper limit of normal value d) Total bile red = 1.5 times the upper limit of normal value e) Glomerular filtration rate eGFR = 60 ml/min 10. For older underage subjects and their legal guardians, it is necessary to have a clear understanding of contraceptive requirements, avoid sexual activity, or use reliable contraceptive measures; 11. Good compliance, willingness of participants and their legal guardians to participate in clinical research on "gene therapy", and regular follow-up. During the follow-up period, they can accurately complete the participant's diary and family members or caregivers can assist the subjects in their progress.
Exclusion criteria
Exclusion criteria: 1. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus deoxyribonucleic acid (HBV DNA), hepatitis C virus antibody (HCV Ab) or hepatitis C virus ribonucleic acid (HCV RNA). Subjects with a history of hepatitis B or hepatitis C should have collected samples twice in the past with an interval of at least 3 months. If the above indicators are negative in both assessments, they will be considered as negative; 2. Receiving antiviral treatment for hepatitis B and hepatitis C; 3. Suffering from coagulation disorders other than hemophilia B; 4. Except for glucocorticoids and immunosuppressants recommended by researchers, any other systemic immunosuppressants are being used within the first 30 days of screening; Known allergies to immunosuppressants such as glucocorticoids and tacrolimus; 5. A history of vaccination within the first 30 days of screening, or a vaccination plan during immunomodulatory therapy; 6. Potential liver diseases, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy, or liver fibrosis = stage 3; Or if nodules or cysts are found through B-ultrasound, or laboratory tests indicate elevated levels of alpha fetoprotein, the researchers have determined that these abnormal conditions have clinical significance and the subjects are not suitable to participate in this study; 7. The planned surgical schedule for the 52 week study period after injection of BBM-H901 injection has been determined; 8. There are acute/chronic infections that may increase the risk due to participating in the experiment, or there are other chronic diseases that the researcher determines are not suitable for participating in the study; 9. Have received gene therapy before screening, or have used other experimental drugs within 4 weeks before screening or within 5 half-lives of the experimental drug (whichever is longer); 10. Any plant preparations (herbal supplements or traditional Chinese medicine derived from plants, minerals, or animals) that may affect liver function were used 4 weeks before or during the study period, except for topical drugs; Or Chinese herbal medicines that may affect this study, as determined by the researcher; 11. The subject has a history of life-threatening bleeding such as intracranial hemorrhage, including situations where the subject is at risk of severe gastrointestinal bleeding due to gastric ulcers; 12. Subjects who suffer from major illnesses or have other conditions that the researcher deems unsuitable to participate in the study; 13. The subjects are family members of research center staff who directly participated in the implementation of the study, family members of research center staff who were otherwise supervised by the researcher, or family members of related company employees who directly participated in the implementation of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of dose limiting toxicity(DLT) events;The incidence of treatment-emergentadverse events (TEAEs) and seriousadverse events (SAEs);Changes in liver function; | — |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetic parameters of Padua-FIX and AAV vectors;Efficacy end points;Safety end points; | — |
Countries
China
Contacts
Public ContactQun Hu
Tongji Medical college of Huazhong University of Science&Technology;Tongji Hospital, Tongji Medical College of Huazhong University of Science&Technology
Outcome results
None listed