[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100603325":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":24,"centralContacts":25,"locations":31,"responsibleParty":104,"collaborators":106,"id":110,"slug":111,"hasResults":112,"nctId":113,"briefTitle":114,"officialTitle":115,"acronym":24,"eligibilityCriteria":116,"healthyVolunteers":112,"sex":117,"minAge":118,"maxAge":119,"enrollmentInfo":120,"targetDuration":24,"studyType":123,"phases":124,"briefSummary":127,"conditions":128,"keywords":130,"overallStatus":34,"whyStopped":24,"lastUpdateSubmitDate":137,"lastUpdatePostDateStruct":138,"startDateStruct":141,"completionDateStruct":143,"leadSponsor":145,"locationsCount":146},{"fullName":5,"class":6},"Mahzi Therapeutics","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"Cohort 1 (MZ-1866)","EXPERIMENTAL","Participants ages 12-25 years",[13],"Genetic: MZ-1866",{"label":15,"type":10,"description":16,"interventionNames":17},"Cohort 2 (MZ-1866)","Participants aged 2-11 years",[13],[19],{"type":20,"name":21,"description":22,"armGroupLabels":23,"otherNames":24},"GENETIC","MZ-1866","AAV-9 gene therapy delivered by intracerebroventricular injection",[9,15],null,[26],{"name":27,"role":28,"phone":29,"phoneExt":24,"email":30},"Emily Radomile","CONTACT","1-844-446-2494","clinicaltrials@mahzi.com",[32,53,70,87],{"facility":33,"status":34,"city":35,"state":36,"zip":37,"country":38,"countryCode":39,"cosmosGeoPoint":40,"geoPoint":45,"contacts":46},"UCSF Benioff Children's Hospital Oakland","RECRUITING","Oakland","California","94609","United States","US",{"type":41,"coordinates":42},"Point",[43,44],-122.2708,37.80437,{"lat":44,"lon":43},[47,50],{"name":48,"role":28,"phone":24,"phoneExt":24,"email":49},"Matt Thura","matt.thura@ucsf.edu",{"name":51,"role":52,"phone":24,"phoneExt":24,"email":24},"Alex Fay, M.D., Ph.D.","PRINCIPAL_INVESTIGATOR",{"facility":54,"status":34,"city":55,"state":56,"zip":57,"country":38,"countryCode":39,"cosmosGeoPoint":58,"geoPoint":62,"contacts":63},"Children's Hospital Colorado","Aurora","Colorado","80045",{"type":41,"coordinates":59},[60,61],-104.83192,39.72943,{"lat":61,"lon":60},[64,68],{"name":65,"role":28,"phone":66,"phoneExt":24,"email":67},"Sky Gould","750-777-1583","sky.gould@childrenscolorado.org",{"name":69,"role":52,"phone":24,"phoneExt":24,"email":24},"Diana Walleigh, MD",{"facility":71,"status":72,"city":73,"state":74,"zip":75,"country":38,"countryCode":39,"cosmosGeoPoint":76,"geoPoint":80,"contacts":81},"Rush University Medical Center","NOT_YET_RECRUITING","Chicago","Illinois","60612",{"type":41,"coordinates":77},[78,79],-87.65005,41.85003,{"lat":79,"lon":78},[82,85],{"name":83,"role":28,"phone":24,"phoneExt":24,"email":84},"Tara Khandelwal","Mehr_Khandelwal@rush.edu",{"name":86,"role":52,"phone":24,"phoneExt":24,"email":24},"Elizabeth Berry-Kravis, M.D., Ph.D.",{"facility":88,"status":34,"city":89,"state":24,"zip":24,"country":90,"countryCode":91,"cosmosGeoPoint":92,"geoPoint":96,"contacts":97},"Sheba Medical Center","Tel Litwinsky","Israel","IL",{"type":41,"coordinates":93},[94,95],34.84588,32.05096,{"lat":95,"lon":94},[98,102],{"name":99,"role":28,"phone":100,"phoneExt":24,"email":101},"Ester Frank","972-52-629-0473","Ester.Frank@sheba.health.gov.il",{"name":103,"role":52,"phone":24,"phoneExt":24,"email":24},"Gali Heimer, M.D., Ph.D.",{"type":105,"investigatorFullName":24,"investigatorTitle":24,"investigatorAffiliation":24,"oldNameTitle":24,"oldOrganization":24},"SPONSOR",[107],{"name":108,"class":109},"California Institute for Regenerative Medicine (CIRM)","OTHER","100603325","phase-1-phase-12-study-of-mz-1866-an-aav-9-gene-therapy-delivered-by-intracerebroventricular-injection-to-participants-with-pitt-hopkins-syndrome-100603325",false,"NCT07135050","Phase 1\u002F2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome","Phase 1\u002F2 First-in-Human Study to Evaluate the Safety, Tolerability, and Efficacy of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome","Inclusion Criteria:\n\n* The participant has a TCF4 gene mutation confirmed as \"pathogenic\" or \"likely pathogenic\" by whole exome sequencing, whole genome sequencing, gene panel, single gene testing, or microarray, performed at an accredited laboratory\n* Clinical phenotype consistent with Pitt Hopkins Syndrome, in the opinion of the investigator\n* The participant, or the participant's parent or legal guardian, is willing to provide access to prior medical records for the collection of demographics and diagnostic and treatment history\n\nExclusion Criteria:\n\n* A deletion that includes the TCF4 gene that is over 12 Mbp in size\n* Another genetic mutation or clinical comorbidity not associated with Pitt Hopkins Syndrome that could potentially confound interpretation of the study data\n* A central nervous system structural or vascular abnormality that is a contraindication to the ICV administration procedure, including but not limited to: signs or symptoms of increased intracranial pressure, history of a space-occupying lesion, or presence of a ventricular shunt that would preclude ICV procedures or safety assessments, or increase risk to the participant\n* Not able to receive prophylactic corticosteroids due to a medical contraindication or participant has a history of a condition that could worsen with corticosteroid therapy as assessed and determined by the Investigator\n* Not able to undergo MRI procedures\n* Cannot be anesthetized for the ICV injection","ALL","2 Years","25 Years",{"count":121,"type":122},12,"ESTIMATED","INTERVENTIONAL",[125,126],"PHASE1","PHASE2","The goal of this clinical trial is to learn if MZ-1866 is a safe and tolerable treatment for children and adults with Pitt Hopkins Syndrome.\n\nTo evaluate the safety of MZ-1866, the following will be evaluated:\n\n* frequency and severity of adverse events\n* physical exam, laboratory results and electrocardiogram findings\n\nParticipants will:\n\n* receive a single dose of MZ-1866 by intracerebroventricular injection\n* be seen by the study physician and site staff periodically to assess changes to their health status\n* be periodically evaluated using neurodevelopmental tools\n\nCaregivers will:\n\n* be interviewed periodically about the health status and development of the participant\n* keep diaries and complete periodic questionnaires regarding participant symptoms",[129],"Pitt Hopkins Syndrome",[21,131,132,133,134,135,136],"AAV9 Gene Therapy","Gene Therapy","ICV","Intracerebroventricular","TCF4","PTHS","2026-05-07",{"date":139,"type":140},"2026-05-11","ACTUAL",{"date":142,"type":140},"2025-12-19",{"date":144,"type":122},"2029-07",{"name":5,"class":6},4]