Ignite Creation Date:
2025-12-25 @ 3:28 AM
Ignite Modification Date:
2025-12-26 @ 2:08 AM
Study NCT ID:
NCT05224505
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-06
First Post:
2021-12-15
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C564612', 'term': 'Muscular Dystrophy, Limb-Girdle, Type 2I'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Single IV administration of ATA-100, open label, 2 sequential cohorts'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 6}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2022-09-01', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2029-09-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-01', 'studyFirstSubmitDate': '2021-12-15', 'studyFirstSubmitQcDate': '2022-02-03', 'lastUpdatePostDateStruct': {'date': '2025-10-06', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2022-02-04', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-02-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and tolerability', 'timeFrame': 'Baseline through 12 months', 'description': 'Incidence of Adverse Events and significant laboratory changes'}], 'secondaryOutcomes': [{'measure': 'Combined endpoint (global testing)', 'timeFrame': 'Baseline through 12 months', 'description': 'Change in North Star Assessment for musculary Dystrophy (NSAD) and Forced Vital capacity (FVC %) from baseline'}, {'measure': 'Timed Up and Go (TUG) test', 'timeFrame': 'Baseline through 12 months', 'description': 'Secondary endpoint'}, {'measure': 'Change from baseline in velocity as measured by 10MWT', 'timeFrame': 'Baseline through 12 months', 'description': '10-meter walk test'}, {'measure': '2-minute walk distance test', 'timeFrame': 'Baseline through 12 months', 'description': 'Secondary endpoint'}, {'measure': 'Cardiac MRI', 'timeFrame': 'Baseline through 12 months', 'description': 'To measure cardiac function (left ejection fraction)'}, {'measure': 'Muscle MRI', 'timeFrame': 'Baseline through 12 months', 'description': 'To measure change from baseline in fat repartition fraction in thigh and leg skeletal muscles'}, {'measure': 'Muscle Biopsy', 'timeFrame': 'Baseline through 12 months', 'description': 'Quantification of FKRP positive muscle fibers'}, {'measure': 'Muscle Biopsy', 'timeFrame': 'Baseline through 12 months', 'description': 'Percentage of glycosylation'}, {'measure': 'Patient reported outcome and quality of life assessment', 'timeFrame': 'Baseline through 12 months', 'description': 'Quality of Life in genetic Neuromuscular Disease (QoL-gNMD), with a range from 0 to 78, the higher the score the worse the quality of life'}, {'measure': 'Patient reported outcome and quality of life assessment', 'timeFrame': 'Baseline through 12 months', 'description': 'ACTIVLIM, scale measuring level of limitation in performing daily activities (total score ranging from 0 to 44, with the lower score the highest limitation)'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['LGMDR9']}, 'descriptionModule': {'briefSummary': 'Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up', 'detailedDescription': 'Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.\n\nThis study is an open-label dose escalation phase with long-term follow-up (LTFU) period.\n\nTwo dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.\n\nAll subjects will be followed for up to 5 years after active IMP (ATA-100) administration.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '99 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 1\\. Female and male ambulant patients\n* 2\\. Patients ≥ 16 years old\n* 3\\. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations\n* 4\\. Moderate diaphragmatic muscle impairment\n\nExclusion Criteria:\n\n* 1\\. Detectable serum neutralizing antibodies against AAV9\n* 2\\. Cardiomyopathy'}, 'identificationModule': {'nctId': 'NCT05224505', 'briefTitle': 'ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9', 'organization': {'class': 'INDUSTRY', 'fullName': 'Atamyo Therapeutics'}, 'officialTitle': 'A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)', 'orgStudyIdInfo': {'id': 'ATA-001-FKRP'}, 'secondaryIdInfos': [{'id': '2021-004276-33', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': 'Cohort 1: single intravenous injection 9.0E+12 vg/Kg', 'interventionNames': ['Biological: ATA-100 (AAV9 encoding FKRP gene)']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'Cohort 2: single intravenous injection 2.7E+13 vg/Kg', 'interventionNames': ['Biological: ATA-100 (AAV9 encoding FKRP gene)']}], 'interventions': [{'name': 'ATA-100 (AAV9 encoding FKRP gene)', 'type': 'BIOLOGICAL', 'description': 'Single intravenous infusion on Day 0', 'armGroupLabels': ['Cohort 1', 'Cohort 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '2100', 'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'Rigshospitalet, University of Copenhagen Blegdamsvej 9', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'zip': '75013', 'city': 'Paris', 'country': 'France', 'facility': "Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital", 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': 'NE1 4LP', 'city': 'Newcastle upon Tyne', 'country': 'United Kingdom', 'facility': 'Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing', 'geoPoint': {'lat': 54.97328, 'lon': -1.61396}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Atamyo Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}