[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100485864":3},{"organization":4,"armGroups":7,"interventions":22,"overallOfficials":29,"centralContacts":34,"locations":40,"responsibleParty":120,"collaborators":28,"id":122,"slug":28,"hasResults":123,"nctId":124,"briefTitle":125,"officialTitle":126,"acronym":28,"eligibilityCriteria":127,"healthyVolunteers":123,"sex":128,"minAge":129,"maxAge":130,"enrollmentInfo":131,"targetDuration":28,"studyType":134,"phases":135,"briefSummary":137,"conditions":138,"keywords":140,"overallStatus":43,"whyStopped":28,"lastUpdateSubmitDate":163,"lastUpdatePostDateStruct":164,"startDateStruct":167,"completionDateStruct":169,"leadSponsor":171,"locationsCount":172},{"fullName":5,"class":6},"Taysha Gene Therapies, Inc.","INDUSTRY",[8,14,18],{"label":9,"type":10,"description":11,"interventionNames":12},"Part A Cohort 1","EXPERIMENTAL","TSHA-102 Dose Level 1: 5.7×10¹⁴ total vector genomes (vg). Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 1 (fully enrolled, 2 participants).",[13],"Genetic: TSHA-102",{"label":15,"type":10,"description":16,"interventionNames":17},"Part A Cohort 2","TSHA-102 Dose Level 2: 1.0×10¹⁵ total vector genomes (vg)\n\nParticipants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (fully enrolled, 4 participants).",[13],{"label":19,"type":10,"description":20,"interventionNames":21},"Part B Pivotal Cohort","TSHA-102 at Selected Dose (Dose Level 2): 1.0 × 10¹⁵ total vector genomes (vg)\n\nParticipants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (1.0 × 10¹⁵).",[13],[23],{"type":24,"name":25,"description":26,"armGroupLabels":27,"otherNames":28},"GENETIC","TSHA-102","TSHA-102 is a recombinant, non-replicating, self-complementary AAV9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration.",[9,15,19],null,[30],{"name":31,"affiliation":32,"role":33},"Medical Monitor, M.D.","Taysha Gene Therapies","STUDY_DIRECTOR",[35],{"name":36,"role":37,"phone":38,"phoneExt":28,"email":39},"Taysha Gene Therapies Medical Information","CONTACT","833-489-8742","medinfo@tayshagtx.com",[41,59,73,86,96,110],{"facility":42,"status":43,"city":44,"state":45,"zip":46,"country":47,"cosmosGeoPoint":48,"geoPoint":53,"contacts":54},"UC San Diego","RECRUITING","La Jolla","California","92093","United States",{"type":49,"coordinates":50},"Point",[51,52],-117.2742,32.84727,{"lat":52,"lon":51},[55],{"name":56,"role":37,"phone":57,"phoneExt":28,"email":58},"Karen Ditslear, M.S.","858-246-2288","kditslear@ucsd.edu",{"facility":60,"status":43,"city":61,"state":62,"zip":63,"country":47,"cosmosGeoPoint":64,"geoPoint":68,"contacts":69},"Rush University Medical Center","Chicago","Illinois","60612",{"type":49,"coordinates":65},[66,67],-87.65005,41.85003,{"lat":67,"lon":66},[70],{"name":71,"role":37,"phone":28,"phoneExt":28,"email":72},"Milana Milic","Milana_Milic@rush.edu",{"facility":74,"status":43,"city":75,"state":76,"zip":77,"country":47,"cosmosGeoPoint":78,"geoPoint":82,"contacts":83},"Boston Children's Hospital","Boston","Massachusetts","02115",{"type":49,"coordinates":79},[80,81],-71.05977,42.35843,{"lat":81,"lon":80},[84],{"name":28,"role":37,"phone":28,"phoneExt":28,"email":85},"RettResearch@childrens.harvard.edu",{"facility":87,"status":43,"city":88,"state":89,"zip":90,"country":47,"cosmosGeoPoint":91,"geoPoint":95,"contacts":28},"Washington University, St. Louis","St Louis","Missouri","63110",{"type":49,"coordinates":92},[93,94],-90.19789,38.62727,{"lat":94,"lon":93},{"facility":97,"status":43,"city":98,"state":99,"zip":100,"country":47,"cosmosGeoPoint":101,"geoPoint":105,"contacts":106},"UT Southwestern Children's Medical Center","Dallas","Texas","75930",{"type":49,"coordinates":102},[103,104],-96.80667,32.78306,{"lat":104,"lon":103},[107],{"name":28,"role":37,"phone":108,"phoneExt":28,"email":109},"214-456-2464","PNTRCstudyrecruitment@utsouthwestern.edu",{"facility":111,"status":43,"city":112,"state":113,"zip":28,"country":114,"cosmosGeoPoint":115,"geoPoint":119,"contacts":28},"CHU St. Justine","Montreal","Quebec","Canada",{"type":49,"coordinates":116},[117,118],-73.58781,45.50884,{"lat":118,"lon":117},{"type":121,"investigatorFullName":28,"investigatorTitle":28,"investigatorAffiliation":28,"oldNameTitle":28,"oldOrganization":28},"SPONSOR","100485864",false,"NCT05606614","A Phase 1\u002F2\u002F3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)","An Open-label Phase 1\u002F2\u002F3 Study Consisting of a Phase 1\u002F2 Safety and Dose-escalation and Phase 3 Dose-expansion Study to Evaluate Safety and Efficacy of a Single Intrathecal Administration of TSHA-102, an AAV9-Delivered Gene Therapy in Females With Rett Syndrome","Inclusion Criteria:\n\n* Females between the ages of 12 and \\\u003C22 in Part A (closed) and females between the ages of 6 and \\\u003C22 in Part B (pivotal cohort).\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","6 Years","21 Years",{"count":132,"type":133},15,"ESTIMATED","INTERVENTIONAL",[136],"PHASE3","The primary objectives of this study are to evaluate the safety of a single intrathecal (IT) dose of TSHA-102 in females with typical Rett syndrome, to select the TSHA-102 dose with the best benefit\u002Frisk profile based on the totality of safety and efficacy data and to evaluate the efficacy and safety of TSHA-102 at the selected dose.",[139],"Rett Syndrome",[139,141,142,143,144,145,146,147,148,149,150,151,152,153,154,155,25,156,157,158,159,160,161,162],"Neurodevelopmental disorder","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","2025-12-29",{"date":165,"type":166},"2025-12-30","ACTUAL",{"date":168,"type":166},"2023-03-06",{"date":170,"type":133},"2031-06",{"name":5,"class":6},6]