[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100603325":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":24,"centralContacts":25,"locations":31,"responsibleParty":102,"collaborators":104,"id":108,"slug":24,"hasResults":109,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":24,"eligibilityCriteria":113,"healthyVolunteers":109,"sex":114,"minAge":115,"maxAge":116,"enrollmentInfo":117,"targetDuration":24,"studyType":120,"phases":121,"briefSummary":124,"conditions":125,"keywords":127,"overallStatus":34,"whyStopped":24,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":138,"completionDateStruct":140,"leadSponsor":142,"locationsCount":143},{"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",[32,52,69,86],{"facility":33,"status":34,"city":35,"state":36,"zip":37,"country":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":45},"UCSF Benioff Children's Hospital Oakland","RECRUITING","Oakland","California","94609","United States",{"type":40,"coordinates":41},"Point",[42,43],-122.2708,37.80437,{"lat":43,"lon":42},[46,49],{"name":47,"role":28,"phone":24,"phoneExt":24,"email":48},"Matt Thura","matt.thura@ucsf.edu",{"name":50,"role":51,"phone":24,"phoneExt":24,"email":24},"Alex Fay, M.D., Ph.D.","PRINCIPAL_INVESTIGATOR",{"facility":53,"status":34,"city":54,"state":55,"zip":56,"country":38,"cosmosGeoPoint":57,"geoPoint":61,"contacts":62},"Children's Hospital Colorado","Aurora","Colorado","80045",{"type":40,"coordinates":58},[59,60],-104.83192,39.72943,{"lat":60,"lon":59},[63,67],{"name":64,"role":28,"phone":65,"phoneExt":24,"email":66},"Sky Gould","750-777-1583","sky.gould@childrenscolorado.org",{"name":68,"role":51,"phone":24,"phoneExt":24,"email":24},"Diana Walleigh, MD",{"facility":70,"status":71,"city":72,"state":73,"zip":74,"country":38,"cosmosGeoPoint":75,"geoPoint":79,"contacts":80},"Rush University Medical Center","NOT_YET_RECRUITING","Chicago","Illinois","60612",{"type":40,"coordinates":76},[77,78],-87.65005,41.85003,{"lat":78,"lon":77},[81,84],{"name":82,"role":28,"phone":24,"phoneExt":24,"email":83},"Tara Khandelwal","Mehr_Khandelwal@rush.edu",{"name":85,"role":51,"phone":24,"phoneExt":24,"email":24},"Elizabeth Berry-Kravis, M.D., Ph.D.",{"facility":87,"status":34,"city":88,"state":24,"zip":24,"country":89,"cosmosGeoPoint":90,"geoPoint":94,"contacts":95},"Sheba Medical Center","Tel Litwinsky","Israel",{"type":40,"coordinates":91},[92,93],34.84588,32.05096,{"lat":93,"lon":92},[96,100],{"name":97,"role":28,"phone":98,"phoneExt":24,"email":99},"Ester Frank","972-52-629-0473","Ester.Frank@sheba.health.gov.il",{"name":101,"role":51,"phone":24,"phoneExt":24,"email":24},"Gali Heimer, M.D., Ph.D.",{"type":103,"investigatorFullName":24,"investigatorTitle":24,"investigatorAffiliation":24,"oldNameTitle":24,"oldOrganization":24},"SPONSOR",[105],{"name":106,"class":107},"California Institute for Regenerative Medicine (CIRM)","OTHER","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":118,"type":119},12,"ESTIMATED","INTERVENTIONAL",[122,123],"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",[126],"Pitt Hopkins Syndrome",[21,128,129,130,131,132,133],"AAV9 Gene Therapy","Gene Therapy","ICV","Intracerebroventricular","TCF4","PTHS","2026-05-07",{"date":136,"type":137},"2026-05-11","ACTUAL",{"date":139,"type":137},"2025-12-19",{"date":141,"type":119},"2029-07",{"name":5,"class":6},4]