Ignite Creation Date:
2025-12-25 @ 4:11 AM
Ignite Modification Date:
2025-12-26 @ 3:09 AM
Study NCT ID:
NCT05898620
Status:
RECRUITING
Last Update Posted:
2025-12-17
First Post:
2023-06-01
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015518', 'term': 'Rett Syndrome'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D065886', 'term': 'Neurodevelopmental Disorders'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D010335', 'term': 'Pathologic Processes'}], 'ancestors': [{'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D001523', 'term': 'Mental Disorders'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['OUTCOMES_ASSESSOR'], 'maskingDescription': 'Central raters for the acquisition of a developmental milestone/skill from videos are blinded to the intervention and to the timing of the video.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'The study treatment will be delivered via intracerebroventricular (ICV) injection. All study participants will receive the same intervention.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 33}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-06-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2029-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-13', 'studyFirstSubmitDate': '2023-06-01', 'studyFirstSubmitQcDate': '2023-06-01', 'lastUpdatePostDateStruct': {'date': '2025-12-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-06-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Efficacy of NGN-401', 'timeFrame': '52 Weeks', 'description': 'Responders will be defined as participants who:\n\n* Attain a CGI-I score of ≤ 3 ("minimally improved");\n* and gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Typical Rett Syndrome', 'MECP2', 'Rett Disorder', 'Genetic Diseases, Inborn', 'Genetic Diseases, X-Linked', 'Neurodevelopmental Disorders', 'Neurobehavioral Manifestations', 'Neurologic Manifestations', 'Intellectual Disability', 'Nervous System Diseases', 'Pathologic Processes', 'RTT'], 'conditions': ['Rett Syndrome']}, 'referencesModule': {'references': [{'pmid': '40173263', 'type': 'DERIVED', 'citation': 'Ross PD, Gadalla KKE, Thomson SR, Selfridge J, Bahey NG, Benito J, Burstein SR, McMinn R, Bolon B, Hector RD, Cobb SR. Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome. Sci Transl Med. 2025 Apr 2;17(792):eadq3614. doi: 10.1126/scitranslmed.adq3614. Epub 2025 Apr 2.'}, {'pmid': '38723617', 'type': 'DERIVED', 'citation': 'Jagadeeswaran I, Oh J, Sinnett SE. Preclinical Milestones in MECP2 Gene Transfer for Treating Rett Syndrome. Dev Neurosci. 2025;47(2):147-156. doi: 10.1159/000539267. Epub 2024 May 9.'}]}, 'descriptionModule': {'briefSummary': 'This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.', 'detailedDescription': "The pivotal study, Embolden (TM), is a conversion of the phase 1/2 study and is an open-label, baseline-controlled, multicenter, single-arm study designed to assess the efficacy, safety, and tolerability of administration of NGN401, an adeno-associated viral vector serotype 9 (AAV9) using Neurogene's proprietary transgene regulation technology. NGN-401 contains a full-length human MECP2 gene and is designed to express therapeutic levels of the MeCP2 protein while avoiding overexpression.\n\nThe study treatment will be given as a single administration under general anesthesia via intracerebroventricular (ICV) delivery. Each participant will be followed for efficacy and safety for 3 years after treatment, and is expected to enroll in a long-term follow-up study for 12 years."}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Females who are between the ages of ≥4 and ≤10 years for Arms 1 and 2 (Arms closed). Females who are ≥11 years of age or older for Arm 3 (Arm closed). Females who are ≥3 for Arm 4, the pivotal cohort.\n* Diagnosis of typical Rett syndrome with a documented disease-causing mutation in the methyl-CpG-binding protein 2 (MECP2) gene\n* Current anti-epileptic drug regimen has been stable for at least 12 weeks\n* Participant must be in the post-regression stage\n* Participant and caregiver should reside within a 2-hour drive of the study center for at least 3 months following treatment\n* Participant must have never taken trofinetide or have taken trofinetide and discontinued due to tolerability, lack of efficacy, or other reasons. Following NGN-401 dosing, trofinetide may be initiated after a specified time period and with the support of the treating clinician.\n\nExclusion Criteria:\n\n* Normal or near normal hand function\n* Has a current clinically significant condition other than Rett syndrome\n* Presence of a concomitant medical condition that precludes intracerebroventricular administration, or use of anesthetics or immune suppression needed for study related procedures\n\nOther inclusion and exclusion criteria apply.'}, 'identificationModule': {'nctId': 'NCT05898620', 'briefTitle': 'A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Neurogene Inc.'}, 'officialTitle': 'A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)', 'orgStudyIdInfo': {'id': 'RTT-200'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Pediatric 1e15 vg dose (fully enrolled)', 'description': 'Dose Level 1 for ages 4-10 years', 'interventionNames': ['Genetic: NGN-401']}, {'type': 'EXPERIMENTAL', 'label': 'Adolescent/Adult 1e15 vg Dose (fully enrolled)', 'description': 'Dose Level 1 for ages 11 years \\& above', 'interventionNames': ['Genetic: NGN-401']}, {'type': 'EXPERIMENTAL', 'label': 'Pediatric 3e15 vg dose (discontinued)', 'description': 'Dose Level 2 for ages 4-10 years (discontinued)', 'interventionNames': ['Genetic: NGN-401']}, {'type': 'EXPERIMENTAL', 'label': 'Pivotal Cohort', 'description': 'Dose Level 1 for ages 3 and above', 'interventionNames': ['Genetic: NGN-401']}], 'interventions': [{'name': 'NGN-401', 'type': 'GENETIC', 'description': 'NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.', 'armGroupLabels': ['Adolescent/Adult 1e15 vg Dose (fully enrolled)', 'Pediatric 1e15 vg dose (fully enrolled)', 'Pediatric 3e15 vg dose (discontinued)', 'Pivotal Cohort']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'mwhatley@uabmc.edu', 'phone': '205-638-6877'}], 'facility': 'University of Alabama at Birmingham', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '94609', 'city': 'Oakland', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'Grace.Sobrero@ucsf.edu'}], 'facility': "UCSF Benioff Children's Hospital Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'NeurogeneRettStudy@childrenscolorado.org'}], 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '33155', 'city': 'Miami', 'state': 'Florida', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'facility': "Nicklaus Children's Hospital Research Institute", 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'Giulia_DiMarco@rush.edu'}], 'facility': 'Rush University Medical Center', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'facility': 'Kennedy Krieger Institute', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'rettresearch@childrens.harvard.edu', 'phone': '617-355-5230'}], 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10467', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'ADJUKIC@montefiore.org'}], 'facility': 'Montefiore Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '27514', 'city': 'Chapel Hill', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'yulissa_gonzalez@neurology.unc.edu', 'phone': '919-843-3516'}], 'facility': 'UNC at Chapel Hill', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'status': 'NOT_YET_RECRUITING', 'country': 'United States', 'facility': 'Vanderbilt University Medical Center', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'role': 'CONTACT', 'email': 'DL-BCM-CRA-Neurogene401Study@bcm.edu'}], 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'city': 'Sydney', 'state': 'New South Wales', 'status': 'ACTIVE_NOT_RECRUITING', 'country': 'Australia', 'facility': "The Children's Hospital at Westmead", 'geoPoint': {'lat': -33.86785, 'lon': 151.20732}}, {'zip': 'EH16 4TJ', 'city': 'Edinburgh', 'status': 'TERMINATED', 'country': 'United Kingdom', 'facility': 'Royal Hospital for Children and Young People', 'geoPoint': {'lat': 55.95206, 'lon': -3.19648}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'status': 'TERMINATED', 'country': 'United Kingdom', 'facility': 'Manchester University NHS Foundation Trust', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}], 'centralContacts': [{'name': 'Contact Center', 'role': 'CONTACT', 'email': 'medicalinfo@neurogene.com', 'phone': '+1 877-237-5020'}], 'overallOfficials': [{'name': 'Julie Jordan, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Neurogene Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Neurogene Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}