[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100493756":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":26,"locations":36,"responsibleParty":59,"collaborators":20,"id":61,"slug":62,"hasResults":63,"nctId":64,"briefTitle":65,"officialTitle":66,"acronym":20,"eligibilityCriteria":67,"healthyVolunteers":63,"sex":68,"minAge":69,"maxAge":70,"enrollmentInfo":71,"targetDuration":20,"studyType":74,"phases":75,"briefSummary":77,"conditions":78,"keywords":80,"overallStatus":39,"whyStopped":20,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":87,"completionDateStruct":89,"leadSponsor":91,"locationsCount":92},{"fullName":5,"class":6},"Institute of Hematology & Blood Diseases Hospital, China","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"BBM-H901 administration group","EXPERIMENTAL","Subjects will be administered with single dose intravenous infusion of BBM-H901.",[13],"Drug: BBM-H901",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"DRUG","BBM-H901","Single dose intravenous infusion of BBM-H901, an adeno-associated viral (AAV) vector designed to drive expression of an hyper active human factor IX mutant(FIX Padua) transgene in liver. The dose of BBM-H901 is 5x10'12 vg\u002FKg.",[9],null,[22],{"name":23,"affiliation":24,"role":25},"Lei Zhang, MD","Insitute of haematology and blood diseases hospital, chinese academy of medical sciences","PRINCIPAL_INVESTIGATOR",[27,32],{"name":28,"role":29,"phone":30,"phoneExt":20,"email":31},"Feng Xue, MD","CONTACT","+862223909240","xuefeng@ihcams.ac.cn",{"name":33,"role":29,"phone":34,"phoneExt":20,"email":35},"Shuo Chen, BS","+862223909009","Chenshuo@ihcams.ac.cn",[37],{"facility":38,"status":39,"city":40,"state":41,"zip":42,"country":43,"countryCode":44,"cosmosGeoPoint":45,"geoPoint":50,"contacts":51},"Institute of haematology and Blood diseases hospital","RECRUITING","Tianjin","Tianjin Municipality","300020","China","CN",{"type":46,"coordinates":47},"Point",[48,49],117.17667,39.14222,{"lat":49,"lon":48},[52,55,56,57],{"name":23,"role":29,"phone":53,"phoneExt":20,"email":54},"0862223909009","zhanglei1@ihcams.ac.cn",{"name":28,"role":29,"phone":30,"phoneExt":20,"email":31},{"name":23,"role":25,"phone":20,"phoneExt":20,"email":20},{"name":28,"role":58,"phone":20,"phoneExt":20,"email":20},"SUB_INVESTIGATOR",{"type":60,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100493756","phase-1-gene-therapy-for-hemophilia-b-patients-aged-12-18-years-old-100493756",false,"NCT05709288","Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old","A Pilot Study Evaluating the Safety, Tolerability and Efficacy of Gene Therapy With BBM-H901 in Hemophilia B Patients Aged 12-18 Years Old","Inclusion Criteria:\n\n1. Subjects and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent;\n2. Be male and 12≤ age \\\u003C18 years of age, body wight ≥ 50kg;\n3. Have hemophilia B with ≤2 IU\u002FdL (≤2 %) endogenous FIX activity levels as documented by a certified clinical laboratory at the time of screening. If the screening result is \\>2% due to insufficient washout from FIX protein product, then the severity of hemophilia B may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤2% FIX coagulant activity (FIX:C) ;\n4. Had had ≥75 prior exposure days (EDs) to any recombinant and\u002For plasma-derived FIX protein products based on historical data from the subject's record\u002Fhistory;\n5. With ≤ 1:4 neutralizing antibodies and ≤1:200 binding antibodies against BBM-H901 capsid;\n6. Subjects with bleeding episode and\u002F or FIX agents infusion events within 12 weeks prior to screening;\n7. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;\n8. Have no measurable FIX inhibitor as assessed by laboratory; or documented no prior history of FIX inhibitor (family history of inhibitors will not exclude the subject) and no clinical signs or symptoms of decreased response to FIX administration;\n9. Have acceptable laboratory values:\n\n   1. Hemoglobin ≥11 g\u002FdL ;\n   2. Platelets ≥100,000 cells\u002FμL;\n   3. AST, ALT ≤1.5x upper limit of normal at the testing laboratory;\n   4. Bilirubin ≤1.5x ULN ;\n   5. glomerular filtration rate eGFR ≥ 60ml\u002Fmin.\n10. For those subjects with sexual maturity, subject and statutory guardian must know that subjects must agree to use reliable barrier contraception until 52 weeks;\n11. with good compliance to the schedule of visit and fill in the subject diary.\n\nExclusion Criteria:\n\n1. Hepatitis B surface antigen antibody (HBSAg-Ab) or HBV-DNA positive; hepatitis C antibody or HCV-RNA positive;\n2. Currently on antiviral therapy for hepatitis B or C;\n3. With coagulation disorders other than hemophilia B;\n4. Had immunosuppressive therapy other than steroid and other suggested IST agents within 30 days prior to screening;\n5. Had vaccine 30 days prior to screening or have scheduled vaccination plan during the study (up to 52 weeks);\n6. Have significant underlying liver disease, as defined by a preexisting diagnosis of portal hypertension, splenomegaly, encephalopathy, etc; other liver conditions unsuitable to gene therapy judged by investigator;\n7. Have surgery plan within 52 weeks after gene therapy;\n8. Have history of chronic infection or high rish of infection that the Investigator considers to constitute an unacceptable risk;\n9. Had participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the last 12 weeks;\n10. Had any herb that may affect the liver function within 4 weeks prior to screening;\n11. Have history of fatal bleeding episode, eg intracranial hemorrhage, etc;\n12. Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study;","MALE","12 Years","18 Years",{"count":72,"type":73},9,"ESTIMATED","INTERVENTIONAL",[76],"PHASE1","This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU\u002Fdl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.",[79],"Hemophilia B",[81,82],"gene therapy","AAV","2025-02-20",{"date":85,"type":86},"2025-02-21","ACTUAL",{"date":88,"type":86},"2023-03-23",{"date":90,"type":73},"2035-11",{"name":5,"class":6},1]