Ignite Creation Date:
2025-12-24 @ 9:43 PM
Ignite Modification Date:
2025-12-25 @ 7:24 PM
Study NCT ID:
NCT07054632
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-10
First Post:
2025-06-27
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 30}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2023-09-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2029-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-07', 'studyFirstSubmitDate': '2025-06-27', 'studyFirstSubmitQcDate': '2025-06-27', 'lastUpdatePostDateStruct': {'date': '2025-07-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-07-08', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-08-13', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Mobility Test', 'timeFrame': '12 months', 'description': 'Changes in functional vision from baseline, determined by mobility test score'}], 'secondaryOutcomes': [{'measure': 'Full-field Light Sensitivity Threshold (FST) Test', 'timeFrame': '6 months、12 months', 'description': 'Changes in light sensitivity from baseline, assessed by FST in log cd.s/m2'}, {'measure': 'Visual Acuity', 'timeFrame': '6 months、12 months', 'description': 'Changes in visual acuity from baseline, based on the ability to read letters using the Early Treatment Diabetic Retionpathy Study (ETDRS) chart'}, {'measure': 'Mobility Test', 'timeFrame': '6 months', 'description': 'Changes in functional vision from baseline, determined by mobility test score'}, {'measure': 'Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)', 'timeFrame': '12 months', 'description': 'Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Inherited Retinal Dystrophy Associated With RPE65 Mutations']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subject and/or their guardian signing a written informed consent.\n\nDiagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.\n\nSubjects are 6 years of age or older.\n\nVisual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.\n\nExclusion Criteria:\n\n* Prior gene therapy for IRD and other hereditary eye diseases.\n\nPre-existing eye conditions that would interfere with interpretation of study endpoints.\n\nActive intraocular or periocular infections in the study eye.\n\nLacking of sufficient surviving retinal cells.\n\nPrior ocular surgery within six months.\n\nComplicating systemic diseases or clinically significant abnormal baseline laboratory values.\n\nPre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds.\n\nComplicating systemic diseases or clinically significant abnormal baseline laboratory values.'}, 'identificationModule': {'nctId': 'NCT07054632', 'briefTitle': 'Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations', 'organization': {'class': 'INDUSTRY', 'fullName': 'Innostellar Biotherapeutics Co.,Ltd'}, 'officialTitle': 'Efficacy and Safety of Gene Therapy rAAV-RPE65 (LX101) in Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy: a Phase III, Multicenter, Randomized Controlled Trial (STAR)', 'orgStudyIdInfo': {'id': 'INNOSTELLAR-LX101-1 (phase 2)'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Intervention group', 'interventionNames': ['Genetic: LX101']}, {'type': 'NO_INTERVENTION', 'label': 'Control group'}], 'interventions': [{'name': 'LX101', 'type': 'GENETIC', 'description': 'Subretinal administration of LX101 to the study eye', 'armGroupLabels': ['Intervention group']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Guangzhou', 'state': 'Guangdong', 'country': 'China', 'facility': 'Zhongshan Ophthalmic Center, Sun Yat sen University', 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}, {'city': 'Chongqing', 'country': 'China', 'facility': 'Southwest Hospital of AMU', 'geoPoint': {'lat': 29.56026, 'lon': 106.55771}}, {'city': 'Shanghai', 'country': 'China', 'facility': 'Shanghai General Hospital', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}, {'city': 'Tianjin', 'country': 'China', 'facility': 'Tianjin Medical University Ophthalmology Hospital', 'geoPoint': {'lat': 39.14222, 'lon': 117.17667}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Innostellar Biotherapeutics Co.,Ltd', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}