[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100532034":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":24,"locations":30,"responsibleParty":44,"collaborators":19,"id":46,"slug":47,"hasResults":48,"nctId":49,"briefTitle":50,"officialTitle":51,"acronym":19,"eligibilityCriteria":52,"healthyVolunteers":48,"sex":53,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":19,"studyType":59,"phases":60,"briefSummary":62,"conditions":63,"keywords":65,"overallStatus":32,"whyStopped":19,"lastUpdateSubmitDate":67,"lastUpdatePostDateStruct":68,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":76},{"fullName":5,"class":6},"Children's Hospital of Fudan University","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Arm of BBM-F101 injection","EXPERIMENTAL","The dose of BBM-F101 injection will be calculated according to the participant's weight with single intravenous infusion.",[13],"Genetic: BBM-F101 injection",[15],{"type":16,"name":17,"description":11,"armGroupLabels":18,"otherNames":19},"GENETIC","BBM-F101 injection",[9],null,[21],{"name":22,"affiliation":5,"role":23},"Hong Xu, MD,PhD","PRINCIPAL_INVESTIGATOR",[25],{"name":26,"role":27,"phone":28,"phoneExt":19,"email":29},"Qian Shen, MD,PhD","CONTACT","+86 13701923307","shenqian@shmu.edu.cn",[31],{"facility":5,"status":32,"city":33,"state":19,"zip":19,"country":34,"countryCode":35,"cosmosGeoPoint":36,"geoPoint":41,"contacts":42},"RECRUITING","Shanghai","China","CN",{"type":37,"coordinates":38},"Point",[39,40],121.45806,31.22222,{"lat":40,"lon":39},[43],{"name":26,"role":27,"phone":28,"phoneExt":19,"email":29},{"type":45,"investigatorFullName":19,"investigatorTitle":19,"investigatorAffiliation":19,"oldNameTitle":19,"oldOrganization":19},"SPONSOR","100532034","early-phase-1-evaluation-of-the-safety-tolerability-and-efficacy-of-a-gene-therapy-drug-for-the-treatment-of-pediatric-fabry-disease-100532034",false,"NCT06207552","Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease","A Single-arm, Open Label, Single-dose Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-F101 Injection in the Treatment of Pediatric Fabry Disease","Inclusion Criteria:\n\n1. The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed consent; If the participant is \\>= 8 years old, the participant must also agree to participate in the study and sign a written informed consent;\n2. Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;\n3. Males or females aged ≥7 years and \\\u003C18 years old;\n4. Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;\n5. Participants had at least one of the clinical manifestations for Fabry disease;\n6. Acceptable capsid antibody titers;\n7. Acceptable anti α-Gal A antibody titers;\n8. Acceptable laboratory values;\n9. Participant's legal guardian and participant with good cooperation and compliance;\n10. Use of reliable contraception methods during the study for adolescence.\n\nExclusion Criteria:\n\n1. Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA), positive for HIV or syphilis;\n2. Have potential liver diseases;\n3. Heart failure and severe arrhythmias;\n4. Severe allergic reactions for enzyme replacement drugs or other medications;\n5. Acute\u002Fchronic infections;\n6. End-stage renal disease;\n7. Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;\n8. Have received gene therapy or used other investigational drugs within four weeks prior to dosing;\n9. Other conditions that make the participant not eligible for the study according to the investigator.","ALL","7 Years","18 Years",{"count":57,"type":58},6,"ESTIMATED","INTERVENTIONAL",[61],"EARLY_PHASE1","This is a single-arm, open label, single-dose clinical study to evaluate the safety, tolerability and efficacy of BBM-F101 injection in the pediatric Fabry disease participants up to 52 weeks after infusion, and the long-term safety and efficacy of BBM-F101 injection up to 5 years after infusion.\n\nBBM-F101 injection is an adeno-associated virus (AAV) gene therapy product for the treatment of pediatric Fabry disease.",[64],"Fabry Disease",[66],"Fabry","2024-07-07",{"date":69,"type":70},"2024-07-09","ACTUAL",{"date":72,"type":70},"2024-02-20",{"date":74,"type":58},"2029-06",{"name":5,"class":6},1]