Ignite Creation Date:
2025-12-24 @ 3:51 PM
Ignite Modification Date:
2026-01-01 @ 6:39 PM
Study NCT ID:
NCT06088992
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-09-19
First Post:
2023-10-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D057130', 'term': 'Leber Congenital Amaurosis'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D012164', 'term': 'Retinal Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 9}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2023-01-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-09', 'completionDateStruct': {'date': '2028-10-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-09-08', 'studyFirstSubmitDate': '2023-10-12', 'studyFirstSubmitQcDate': '2023-10-12', 'lastUpdatePostDateStruct': {'date': '2024-09-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-10-18', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-10-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence and severity of ocular and systemic adverse events', 'timeFrame': '26 weeks', 'description': 'Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Leber Congenital Amaurosis']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutationsin RPE65 gene.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '8 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or females between 8 and 50 years of age at the time of signing theinformed consent form.\n* Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent.\n* Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations.\n* Ability to perform tests of visual and retinal function.\n* Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected.\n* Acceptable hematology, clinical chemistry, and urine laboratory parameters.\n\nExclusion Criteria:\n\n* OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye.\n* Presence of epiretinal membrane by OCT.\n* Complicating systemic diseases or clinically significant abnormal baselinelaboratory values.\n* Complicating systemic diseases would include those in which the diseaseitself, or the treatment for the disease, can alter ocular function.\n* Prior ocular surgery within six months.\n* Prior gene therapy or oligonucleotide therapy treatments.\n* Any other condition that would not allow the potential subject to completefollow-up examinations during the study and would, in the opinion of theinvestigator, make the potential subject unsuitable for the study.'}, 'identificationModule': {'nctId': 'NCT06088992', 'briefTitle': 'Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)', 'nctIdAliases': ['NCT06064565'], 'organization': {'class': 'OTHER', 'fullName': 'Xinhua Hospital, Shanghai Jiao Tong University School of Medicine'}, 'officialTitle': 'An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety,Tolerability, and Efficacy of Gene Therapy for 2Leber's Congenital Amaurosis with RPE65 Mutation (LCA2)', 'orgStudyIdInfo': {'id': 'HG00401'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HG004', 'interventionNames': ['Genetic: HG004']}], 'interventions': [{'name': 'HG004', 'type': 'GENETIC', 'description': 'Method of Administration: Once unilateralsubretinal injection; The duration of the study isabout 60 weeks for each subject including a 8-weekscreening period, enrollment/baseline visit,treatment visit, and 52 weeks follow-up period.', 'armGroupLabels': ['HG004']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Shanghai', 'state': 'Shanghai Municipality', 'country': 'China', 'facility': 'Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}], 'overallOfficials': [{'name': 'Peiquan Zhao', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Xinhua Hospital affiliated with Shanghai Jiao Tong UniversitySchool of Medicine'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Xinhua Hospital, Shanghai Jiao Tong University School of Medicine', 'class': 'OTHER'}, 'collaborators': [{'name': 'HuidaGene Therapeutics Co., Ltd.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Professor', 'investigatorFullName': 'Peiquan Zhao', 'investigatorAffiliation': 'Xinhua Hospital, Shanghai Jiao Tong University School of Medicine'}}}}