[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100485864":3},{"organization":4,"armGroups":7,"interventions":22,"overallOfficials":29,"centralContacts":34,"locations":40,"responsibleParty":122,"collaborators":28,"id":124,"slug":125,"hasResults":126,"nctId":127,"briefTitle":128,"officialTitle":129,"acronym":28,"eligibilityCriteria":130,"healthyVolunteers":126,"sex":131,"minAge":132,"maxAge":133,"enrollmentInfo":134,"targetDuration":28,"studyType":137,"phases":138,"briefSummary":140,"conditions":141,"keywords":143,"overallStatus":43,"whyStopped":28,"lastUpdateSubmitDate":166,"lastUpdatePostDateStruct":167,"startDateStruct":170,"completionDateStruct":172,"leadSponsor":174,"locationsCount":175},{"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",[41,60,74,87,97,111],{"facility":42,"status":43,"city":44,"state":45,"zip":46,"country":47,"countryCode":48,"cosmosGeoPoint":49,"geoPoint":54,"contacts":55},"UC San Diego","RECRUITING","La Jolla","California","92093","United States","US",{"type":50,"coordinates":51},"Point",[52,53],-117.2742,32.84727,{"lat":53,"lon":52},[56],{"name":57,"role":37,"phone":58,"phoneExt":28,"email":59},"Karen Ditslear, M.S.","858-246-2288","kditslear@ucsd.edu",{"facility":61,"status":43,"city":62,"state":63,"zip":64,"country":47,"countryCode":48,"cosmosGeoPoint":65,"geoPoint":69,"contacts":70},"Rush University Medical Center","Chicago","Illinois","60612",{"type":50,"coordinates":66},[67,68],-87.65005,41.85003,{"lat":68,"lon":67},[71],{"name":72,"role":37,"phone":28,"phoneExt":28,"email":73},"Milana Milic","Milana_Milic@rush.edu",{"facility":75,"status":43,"city":76,"state":77,"zip":78,"country":47,"countryCode":48,"cosmosGeoPoint":79,"geoPoint":83,"contacts":84},"Boston Children's Hospital","Boston","Massachusetts","02115",{"type":50,"coordinates":80},[81,82],-71.05977,42.35843,{"lat":82,"lon":81},[85],{"name":28,"role":37,"phone":28,"phoneExt":28,"email":86},"RettResearch@childrens.harvard.edu",{"facility":88,"status":43,"city":89,"state":90,"zip":91,"country":47,"countryCode":48,"cosmosGeoPoint":92,"geoPoint":96,"contacts":28},"Washington University, St. Louis","St Louis","Missouri","63110",{"type":50,"coordinates":93},[94,95],-90.19789,38.62727,{"lat":95,"lon":94},{"facility":98,"status":43,"city":99,"state":100,"zip":101,"country":47,"countryCode":48,"cosmosGeoPoint":102,"geoPoint":106,"contacts":107},"UT Southwestern Children's Medical Center","Dallas","Texas","75930",{"type":50,"coordinates":103},[104,105],-96.80667,32.78306,{"lat":105,"lon":104},[108],{"name":28,"role":37,"phone":109,"phoneExt":28,"email":110},"214-456-2464","PNTRCstudyrecruitment@utsouthwestern.edu",{"facility":112,"status":43,"city":113,"state":114,"zip":28,"country":115,"countryCode":116,"cosmosGeoPoint":117,"geoPoint":121,"contacts":28},"CHU St. Justine","Montreal","Quebec","Canada","CA",{"type":50,"coordinates":118},[119,120],-73.58781,45.50884,{"lat":120,"lon":119},{"type":123,"investigatorFullName":28,"investigatorTitle":28,"investigatorAffiliation":28,"oldNameTitle":28,"oldOrganization":28},"SPONSOR","100485864","phase-3-a-phase-123-study-of-tsha-102-gene-therapy-in-females-with-rett-syndrome-reveal-pivotal-study-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":135,"type":136},15,"ESTIMATED","INTERVENTIONAL",[139],"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.",[142],"Rett Syndrome",[142,144,145,146,147,148,149,150,151,152,153,154,155,156,157,158,25,159,160,161,162,163,164,165],"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":168,"type":169},"2025-12-30","ACTUAL",{"date":171,"type":169},"2023-03-06",{"date":173,"type":136},"2031-06",{"name":5,"class":6},6]