[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Mahzi Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":50},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"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":20,"type":21},12,"ESTIMATED","INTERVENTIONAL",[24,25],"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",[28],"Pitt Hopkins Syndrome",[30,31,32,33,34,35,36],"MZ-1866","AAV9 Gene Therapy","Gene Therapy","ICV","Intracerebroventricular","TCF4","PTHS","RECRUITING","2026-05-07",{"date":40,"type":41},"2026-05-11","ACTUAL",{"date":43,"type":41},"2025-12-19",{"date":45,"type":21},"2029-07",{"name":47,"class":48},"Mahzi Therapeutics","INDUSTRY",4,""]