Ignite Creation Date:
2025-12-25 @ 5:19 AM
Ignite Modification Date:
2025-12-26 @ 4:26 AM
Study NCT ID:
NCT05567627
Status:
UNKNOWN
Last Update Posted:
2023-11-01
First Post:
2022-09-25
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 6}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-08-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-10', 'completionDateStruct': {'date': '2025-09', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-10-30', 'studyFirstSubmitDate': '2022-09-25', 'studyFirstSubmitQcDate': '2022-09-30', 'lastUpdatePostDateStruct': {'date': '2023-11-01', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-10-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change from baseline glycogen content in muscle tissue', 'timeFrame': '26 and 52 weeks'}, {'measure': 'Change from baseline acid alpha-glucosidase (GAA) enzyme in muscle and blood', 'timeFrame': '26 and 52 weeks'}, {'measure': "Improvement in patient's motor function", 'timeFrame': '52 weeks', 'description': "To evaluate the changes in patient's mobility and physical ability using Hammersmith Infant Neurological Examination (HINE) scores"}, {'measure': 'The viral load of adeno-associated virus (AAV) vector', 'timeFrame': 'At multiple time points from pre-dose through up to 1 years post-dose', 'description': 'To assess the change of AAV vector copy numbers within 52 weeks after administration.'}], 'primaryOutcomes': [{'measure': 'Safety and tolerability over time', 'timeFrame': 'Infusion to the end of study, average 1 year', 'description': 'Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests'}], 'secondaryOutcomes': [{'measure': 'Proportion of patients treated w/ GC301 who were alive and free of ventilator support at 12 months of age;', 'timeFrame': '52 weeks'}, {'measure': 'Changes from baseline Left Ventricular Mass (LVM)', 'timeFrame': '26 and 52 weeks'}, {'measure': 'Changes from baseline creatine kinase (CK)', 'timeFrame': '26 and 52 weeks'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Infantile-onset Pompe Disease']}, 'descriptionModule': {'briefSummary': 'This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed;\n* The patient must be no older than 6 months;\n* The patient must be diagnosed with infantile-onset Pompe disease.\n\nExclusion Criteria:\n\n* Class IV patient based on Modified Ross Heart Failure Classification for Children;\n* Aspartate aminotransferase (AST), alanine aminotransferase (ALT) \\> 3x upper limit of normal (ULN), alkaline phosphatase (ALP) \\> 2x ULN (with the exception of liver abnormalities related to Pompe disease);\n* Patient has severe organ dysfunction, such as liver and kidney failure (Liver failure: patients may have liver failure syndrome, including fatigue, severe gastrointestinal symptoms; clinical examination found prolonged prothrombin time, prothrombin activity less than 40%; Neuropsychiatric symptoms, such as restlessness, changes in personality and behavior, lethargy, coma, etc.; Toxic tympanic bowel, ascites, multiple organ dysfunction, etc.; hyperalbuminemia exceeding 171 μmol/L, hypoalbuminemia. Renal failure: creatinine exceeding 110 μmol/L, or glomerular filtration rate less than 100 mL/min), congenital/acquired encephalopathy, etc.;\n* Patient with congenital organ absence;\n* Patient with primary immunodeficiency;\n* Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;\n* Patient with a history of glucocorticoid allergy；\n* Patient who has participated in a previous gene therapy research trial;\n* Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study."}, 'identificationModule': {'nctId': 'NCT05567627', 'briefTitle': 'Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease', 'organization': {'class': 'OTHER', 'fullName': 'Seventh Medical Center of PLA General Hospital'}, 'officialTitle': 'Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease', 'orgStudyIdInfo': {'id': 'JLJY-GC301-IOPD-003'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Initial dose cohort', 'description': '1.2x10\\^14 vg/kg of GC301 administered via intravenous infusion', 'interventionNames': ['Biological: Genetic: GC301']}], 'interventions': [{'name': 'Genetic: GC301', 'type': 'BIOLOGICAL', 'description': 'GC301, is an adeno-associated virus 9 (AAV9) vector delivering a functional copy of the human GAA gene', 'armGroupLabels': ['Initial dose cohort']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100700', 'city': 'Beijing', 'state': 'Beijing Municipality', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Zhichun Feng, Prof', 'role': 'CONTACT', 'email': 'zhichunfeng81@163.com', 'phone': '+86(10) 66721786'}, {'name': 'Xiaodong Wang, PhD', 'role': 'CONTACT', 'email': 'donnawang@bj-genecradle.com', 'phone': '+86 17801060980'}], 'facility': "Bayi Children's Hospital, Seventh Medical Center, PLA general hospital", 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'centralContacts': [{'name': 'Zhichun Feng', 'role': 'CONTACT', 'email': 'zhichunfeng81@163.com', 'phone': '+86(10) 66721786'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Seventh Medical Center of PLA General Hospital', 'class': 'OTHER'}, 'collaborators': [{'name': 'GeneCradle Therapeutics, Inc', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'SPONSOR'}}}}