Ignite Creation Date:
2025-12-24 @ 11:03 PM
Ignite Modification Date:
2026-01-03 @ 10:36 PM
Study NCT ID:
NCT06567769
Status:
RECRUITING
Last Update Posted:
2025-08-28
First Post:
2024-08-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009084', 'term': 'Mucopolysaccharidosis III'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 9}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-11-21', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2027-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-21', 'studyFirstSubmitDate': '2024-08-20', 'studyFirstSubmitQcDate': '2024-08-20', 'lastUpdatePostDateStruct': {'date': '2025-08-28', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-08-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-06', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidences and characteristics of adverse events', 'timeFrame': 'up to 108 weeks'}], 'secondaryOutcomes': [{'measure': 'Maximum concentration in cerebrospinal fluid (CSF)', 'timeFrame': 'up to 104 weeks', 'description': 'GC1130A PK parameters'}, {'measure': 'Area under the concentration-time curve in CSF', 'timeFrame': 'up to 104 weeks', 'description': 'GC1130A PK parameters'}, {'measure': 'Maximum concentration in serum', 'timeFrame': 'up to 104 weeks', 'description': 'GC1130A PK parameters'}, {'measure': 'Area under the concentration-time curve in serum', 'timeFrame': 'up to 104 weeks', 'description': 'GC1130A PK parameters'}, {'measure': 'Change from baseline in CSF heparan sulfate concentration', 'timeFrame': 'up to 104 weeks'}, {'measure': 'Change from baseline in serum heparan sulfate concentration', 'timeFrame': 'up to 104 weeks'}, {'measure': 'Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF', 'timeFrame': 'up to 104 weeks'}, {'measure': 'Incidence of anti-drug and neutralizing antibodies of GC1130A in serum', 'timeFrame': 'up to 104 weeks'}, {'measure': 'Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)', 'timeFrame': 'up to 104 weeks'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Mucopolysaccharidosis Type III Subtype A', 'MPS IIIA'], 'conditions': ['Sanfilippo Syndrome Type A']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '12 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants with documented MPS IIIA diagnosis\n* Participants aged ≥ 12 months and ≤ 18 years\n\nExclusion Criteria:\n\n* Participants with significant non-MPS IIIA related central nervous system impairment\n* Participants with previous complication from intraventricular drug administration\n* Participants with contraindications for MRI scans and for neurosurgery\n* Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study\n* Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy'}, 'identificationModule': {'nctId': 'NCT06567769', 'briefTitle': 'Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)', 'organization': {'class': 'INDUSTRY', 'fullName': 'GC Biopharma Corp'}, 'officialTitle': 'A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)', 'orgStudyIdInfo': {'id': 'GC1130_MPS3A_P0101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'interventionNames': ['Drug: GC1130A']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'interventionNames': ['Drug: GC1130A']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'interventionNames': ['Drug: GC1130A']}], 'interventions': [{'name': 'GC1130A', 'type': 'DRUG', 'description': 'ICV injection', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 3']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mariam Samara', 'role': 'CONTACT', 'email': 'Mariam.Samara@ucsf.edu', 'phone': '510-428-3885', 'phoneExt': '4785'}, {'name': 'Paul Harmatz, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "UCSF Benioff Children's Hospital", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Brenda Diethelm-Okita', 'role': 'CONTACT', 'email': 'dieth001@umn.edu', 'phone': '612-625-1594'}, {'name': 'Melissa Fetterley', 'role': 'CONTACT', 'email': 'Melissa.Fetterley@fairview.org', 'phone': '612-672-5151'}, {'name': 'Chester Whitley, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'University of Minnesota', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'city': 'Setagaya City', 'state': 'Tokyo', 'status': 'RECRUITING', 'country': 'Japan', 'contacts': [{'name': 'Motomichi Kosuga, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'National Center for Child Health and Development', 'geoPoint': {'lat': 35.64188, 'lon': 139.64715}}, {'city': 'Suwon', 'state': 'Gyeongi-do', 'status': 'RECRUITING', 'country': 'South Korea', 'contacts': [{'name': 'Bora Lim', 'role': 'CONTACT', 'email': '115694@aumc.ac.kr', 'phone': '+82312194404'}, {'name': 'Young Bae Sohn, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Ajou University Medical Center', 'geoPoint': {'lat': 37.29111, 'lon': 127.00889}}, {'city': 'Seoul', 'status': 'RECRUITING', 'country': 'South Korea', 'contacts': [{'name': 'Minji Im, MD', 'role': 'CONTACT', 'email': 'mj33.im@samsung.com', 'phone': '+82234103757'}, {'name': 'Sung Yoon Cho, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Samsung Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}], 'centralContacts': [{'name': 'GC Biopharma Corp.', 'role': 'CONTACT', 'email': 'mps3a_clinicaltrial@gccorp.com', 'phone': '+82312609300'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'GC Biopharma Corp', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}