[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100629891":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":26,"locations":32,"responsibleParty":49,"collaborators":20,"id":51,"slug":20,"hasResults":52,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":56,"eligibilityCriteria":57,"healthyVolunteers":52,"sex":58,"minAge":59,"maxAge":60,"enrollmentInfo":61,"targetDuration":20,"studyType":64,"phases":65,"briefSummary":67,"conditions":68,"keywords":70,"overallStatus":35,"whyStopped":20,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":102},{"fullName":5,"class":6},"Taysha Gene Therapies, Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Treatment","EXPERIMENTAL","Participants receive a single intrathecal (IT) administration of TSHA-102 at 1.0 × 10¹⁵ total vector genomes (vg) adjusted for the participant's brain volume.",[13],"Genetic: TSHA-102",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"GENETIC","TSHA-102","TSHA-102 is a recombinant, non-replicating, self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.",[9],null,[22],{"name":23,"affiliation":24,"role":25},"Medical Monitor, M.D.","Taysha Gene Therapies","STUDY_DIRECTOR",[27],{"name":28,"role":29,"phone":30,"phoneExt":20,"email":31},"Taysha Gene Therapies Medical Information","CONTACT","833-489-8742","medinfo@tayshagtx.com",[33],{"facility":34,"status":35,"city":36,"state":37,"zip":38,"country":39,"cosmosGeoPoint":40,"geoPoint":45,"contacts":46},"Boston Children's Hospital","RECRUITING","Boston","Massachusetts","02115","United States",{"type":41,"coordinates":42},"Point",[43,44],-71.05977,42.35843,{"lat":44,"lon":43},[47],{"name":20,"role":29,"phone":20,"phoneExt":20,"email":48},"RettResearch@childrens.harvard.edu",{"type":50,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100629891",false,"NCT07480564","Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to \u003C4 Years With Rett Syndrome","ASPIRE Study: A Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy, for the Treatment of Pediatric Females Aged >2 to \u003C4 Years With Rett Syndrome","ASPIRE","Inclusion Criteria:\n\n* Pediatric females between the ages of 2 and less than 4 years old.\n* Participant has a clinical diagnosis of classic\u002Ftypical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function.\n* Participants must be willing to receive blood or blood products for the treatment of an AE if medically needed.\n* Participants and parent\u002Fcaregiver must agree to reside within easy access to the study site prior to the baseline visit and at least 3 months after TSHA-102 treatment.\n\nExclusion Criteria:\n\n* Participant has another neurodevelopmental disorder independent of the MECP2 loss-of-function mutation, or any other genetic syndrome with a progressive course.\n* Participant has a history of brain injury that causes neurological problems or had grossly abnormal psychomotor development in the first 6 months of life.\n* Participant has a diagnosis of atypical Rett syndrome or a MECP2 gene mutation that does not cause Rett syndrome.\n* Participant requires invasive ventilatory support.\n\nNote: Other protocol defined inclusion\u002Fexclusion criteria may apply","FEMALE","2 Years","3 Years",{"count":62,"type":63},3,"ESTIMATED","INTERVENTIONAL",[66],"PHASE3","The primary objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of a single intrathecal (IT) dose of TSHA-102 in pediatric females with typical Rett syndrome.",[69],"Rett Syndrome",[71,72,73,74,75,76,77,78,79,80,81,82,83,84,85,17,86,87,88,89,90,91,92],"Neurodevelopmental Disorders","Rett","MECP2","AAV9","Typical Rett Syndrome","Classic Rett Syndrome","RTT","Rett Disorder","Retts","MECP2-Related Disorder","Gene Therapy","Intrathecal Administration","Genetic Diseases, X-Linked","Nervous System Diseases","Developmental Regression","miRARE","Self-complementary Vector","Neurologic Manifestations","Intellectual Disability","Pathologic Process","X-Linked Intellectual Disability","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","2026-05-20",{"date":95,"type":96},"2026-05-22","ACTUAL",{"date":98,"type":96},"2026-05-08",{"date":100,"type":63},"2031-06",{"name":5,"class":6},1]