Ignite Creation Date:
2025-12-24 @ 4:50 PM
Ignite Modification Date:
2026-01-02 @ 10:39 AM
Study NCT ID:
NCT07135050
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-12
First Post:
2025-08-04
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C537403', 'term': 'Pitt-Hopkins syndrome'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 12}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2026-01', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2029-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-10', 'studyFirstSubmitDate': '2025-08-04', 'studyFirstSubmitQcDate': '2025-08-13', 'lastUpdatePostDateStruct': {'date': '2025-12-12', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-08-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To evaluate the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome', 'timeFrame': 'Week 104', 'description': 'Frequency and severity of adverse events of special interest'}], 'secondaryOutcomes': [{'measure': 'To evaluate the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome', 'timeFrame': 'Week 104', 'description': 'Frequency and severity of adverse events'}, {'measure': 'To further assess the safety and tolerability of MZ-1866 in participants with Pitt Hopkins Syndrome', 'timeFrame': 'Week 104', 'description': 'Frequency of clinically significant physical examination findings, vital signs, laboratory tests and electrocardiograms that are considered outside normal range.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['MZ-1866', 'AAV9 Gene Therapy', 'Gene Therapy', 'ICV', 'Intracerebroventricular', 'TCF4', 'PTHS'], 'conditions': ['Pitt Hopkins Syndrome']}, 'descriptionModule': {'briefSummary': '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'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '25 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': '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'}, 'identificationModule': {'nctId': 'NCT07135050', 'briefTitle': 'Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Mahzi Therapeutics'}, 'officialTitle': 'Phase 1/2 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', 'orgStudyIdInfo': {'id': 'MZ1866-CL101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1 (MZ-1866)', 'description': 'Participants ages 12-25 years', 'interventionNames': ['Genetic: MZ-1866']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2 (MZ-1866)', 'description': 'Participants aged 2-11 years', 'interventionNames': ['Genetic: MZ-1866']}], 'interventions': [{'name': 'MZ-1866', 'type': 'GENETIC', 'description': 'AAV-9 gene therapy delivered by intracerebroventricular injection', 'armGroupLabels': ['Cohort 1 (MZ-1866)', 'Cohort 2 (MZ-1866)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'contacts': [{'name': 'Matt Thura', 'role': 'CONTACT', 'email': 'matt.thura@ucsf.edu'}, {'name': 'Alex Fay, M.D., Ph.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "UCSF Benioff Children's Hospital Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'contacts': [{'name': 'Milana Milic', 'role': 'CONTACT', 'email': 'milana_milic@rush.edu'}, {'name': 'Elizabeth Berry-Kravis, M.D., Ph.D.', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Rush University Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}], 'centralContacts': [{'name': 'Emily Radomile', 'role': 'CONTACT', 'email': 'clinicaltrials@mahzi.com', 'phone': '1-844-446-2494'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Mahzi Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}