[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100418448":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":26,"locations":34,"responsibleParty":56,"collaborators":20,"id":58,"slug":59,"hasResults":60,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":64,"eligibilityCriteria":65,"healthyVolunteers":60,"sex":66,"minAge":67,"maxAge":20,"enrollmentInfo":68,"targetDuration":20,"studyType":71,"phases":72,"briefSummary":74,"conditions":75,"keywords":20,"overallStatus":36,"whyStopped":20,"lastUpdateSubmitDate":78,"lastUpdatePostDateStruct":79,"startDateStruct":82,"completionDateStruct":84,"leadSponsor":86,"locationsCount":87},{"fullName":5,"class":6},"Institute of Hematology & Blood Diseases Hospital, China","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Treatment group","EXPERIMENTAL","Arm of GS001",[13],"Genetic: Injection of GS001",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"GENETIC","Injection of GS001","Patients will be enrolled sequentially every 3 weeks or more between cohorts. Dose escalation may occur after a single patient has been safely dosed if the resulting FVIII activity at Week 3 is \\\u003C 5 IU\u002FdL.The dose levels are as follows:\n\n1. 2×10\\^12 vg\u002Fkg\n2. 6×10\\^12vg\u002Fkg or other recommended doses\n3. 2×10\\^13 vg\u002Fkg or other recommended doses",[9],null,[22],{"name":23,"affiliation":24,"role":25},"Lei Zhang, MD","Chinese Academy of Medical Science and Blood Disease Hospital","PRINCIPAL_INVESTIGATOR",[27,32],{"name":28,"role":29,"phone":30,"phoneExt":20,"email":31},"Wei Liu, MD","CONTACT","+82223909240","liuwei1@ihcams.ac.cn",{"name":23,"role":29,"phone":30,"phoneExt":20,"email":33},"zhanglei1@ihcams.ac.cn",[35],{"facility":24,"status":36,"city":37,"state":38,"zip":39,"country":40,"countryCode":41,"cosmosGeoPoint":42,"geoPoint":47,"contacts":48},"RECRUITING","Tianjin","Tianjin Municipality","300020","China","CN",{"type":43,"coordinates":44},"Point",[45,46],117.17667,39.14222,{"lat":46,"lon":45},[49,52],{"name":50,"role":29,"phone":51,"phoneExt":20,"email":33},"Shuo Chen, MD","+862223909240",{"name":53,"role":29,"phone":54,"phoneExt":20,"email":55},"Jun L Liu, MD","+862223909095","gcp@ihcams.ac.cn",{"type":57,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100418448","gene-therapy-for-chinese-hemophilia-a-100418448",false,"NCT04728841","Gene Therapy for Chinese Hemophilia A","Clinical Exploration of Clinical Exploration Adeno-associated Virus Vector Expression of Human Coagulation Factor VIII Gene Therapy for Hemophilia A","HA","Inclusion Criteria:\n\n1. Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws;\n2. Male subjects and ≥ 18 years of age;\n3. Have hemophilia A with ≤1 IU\u002FdL (≤1%) endogenous FVIII activity levels at the time of screening. If the screening result is \\>1% due to previous treatment with FVIII product, then it may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤1% FVIII activity levels ;\n4. No history of hypersensitivity or anaphylaxis associated with FVIII product administration;\n5. Have no measurable FVII inhibitor as assessed by laboratory two times that were at least one week apart; or documented no prior history of FVIII inhibitor after 150 EDs and no clinical signs or symptoms of decreased response to FVIII infusion ;\n6. Have acceptable laboratory values sampled at screening and repeated prior to Day 0; A. Hemoglobin ≥ 11 g\u002FdL; B. Platelets ≥ 100 x 10\\^9\u002FL; C. AST, ALT, alkaline phosphatase ≤ 1.25 upper limit of normal (ULN); D. Bilirubin ≤ 1.25 ULN; E. Creatinine ≤ 2 mg\u002FdL.\n7. Agree to use reliable barrier contraception until the end of the 52 weeks observation period, and three consecutive semen samples are negative for vector sequences after GS001 infusion.\n\nExclusion Criteria:\n\n1. Have Hepatitis B, hepatitis C or HBsAg, HCVAb, HBV-DNA, HCV-RNA are positive and have clinical significance. Both natural clearers and those who have cleared HCV on antiviral therapy are deemed eligible;\n2. Currently Receiving antiviral therapy for hepatitis B and C;\n3. Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;\n4. Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the past 30 days;\n5. The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator;\n6. History of arterial or venous thromboembolic events (e.g., deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolism);\n7. Known inherited or acquired thrombophilia, including conditions associated with increased risk of thromboembolism, such as atrial fibrillation;\n8. Major surgery planned in 1 year period following the infusion with GS001;\n9. Hypersensitivity to the study vector;\n10. Have clinically major diseases or any other unspecified conditions that, in the opinion of the Investigator, makes the subject unsuitable for participating in the study;\n11. Patients who are unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;\n12. Evidence of other bleeding disorders not associated with hemophilia A.","MALE","18 Years",{"count":69,"type":70},12,"ESTIMATED","INTERVENTIONAL",[73],"NA","IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \\\u003C1 IU\u002Fdl residual FVIII levels.",[76,77],"Hemophilia A","Gene Therapy","2025-07-31",{"date":80,"type":81},"2025-08-01","ACTUAL",{"date":83,"type":81},"2021-03-04",{"date":85,"type":70},"2028-07-31",{"name":5,"class":6},1]