Ignite Creation Date:
2025-12-24 @ 3:02 PM
Ignite Modification Date:
2026-02-25 @ 11:01 PM
Study NCT ID:
NCT01625559
Status:
COMPLETED
Last Update Posted:
2024-04-10
First Post:
2012-06-18
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-02', 'completionDateStruct': {'date': '2015-04', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-04-08', 'studyFirstSubmitDate': '2012-06-18', 'studyFirstSubmitQcDate': '2012-06-19', 'lastUpdatePostDateStruct': {'date': '2024-04-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2012-06-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-04', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'safety and tolerance of transplantation', 'timeFrame': '18 months', 'description': 'The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:\n\n1. Any grade 2 (NCI grading system) or greater adverse event related to the cell product\n2. Any evidence that the cells are contaminated with an infectious agent\n3. Any evidence that the cells show tumorigenic potential'}], 'secondaryOutcomes': [{'measure': 'Evidence of successful engraftment', 'timeFrame': '18 months', 'description': 'Evidence of successful engraftmentEvidence of successful engraftment will consist of:\n\nStructural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location Electroretinographic evidence (mfERG) showing enhanced activity in the implant location'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ["Stargardt's Macular Dystrophy"]}, 'referencesModule': {'references': [{'pmid': '32727729', 'type': 'DERIVED', 'citation': 'Sung Y, Lee MJ, Choi J, Jung SY, Chong SY, Sung JH, Shim SH, Song WK. Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients. Br J Ophthalmol. 2021 Jun;105(6):829-837. doi: 10.1136/bjophthalmol-2020-316225. Epub 2020 Jul 29.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD', 'detailedDescription': '* to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD\n* to evaluate the preliminary efficacy of MA09-hPRE in patients with SMD'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '20 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adult male or female over 20 years of age.\n* Clinical diagnosis of advanced SMD.\n* The visual acuity of the eye to receive the transplant will be no better than hand movement.\n* The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.\n\nExclusion Criteria:\n\n* History of malignancy.\n* History of myocardial infarction in previous 12 months.\n* History of diabetes mellitus.\n* Any immunodeficiency.\n* Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.\n* Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.\n* Current participation in any other clinical trial.\n* Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.\n* Any other sight-threatening ocular disease.\n* Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months'}, 'identificationModule': {'nctId': 'NCT01625559', 'briefTitle': "Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)", 'organization': {'class': 'INDUSTRY', 'fullName': 'CHABiotech CO., Ltd'}, 'officialTitle': "A Phase I, Open-Label, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy(SMD)", 'orgStudyIdInfo': {'id': 'CHA_CTP_0903'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'MA09-hRPE administration', 'description': 'Biological: MA09-hRPE Cellular therapy', 'interventionNames': ['Biological: MA09-hRPE']}], 'interventions': [{'name': 'MA09-hRPE', 'type': 'BIOLOGICAL', 'description': 'MA09-hRPE cells', 'armGroupLabels': ['MA09-hRPE administration']}]}, 'contactsLocationsModule': {'locations': [{'zip': '463-712', 'city': 'Seongnam-si', 'state': 'Gyeonggi-do', 'country': 'South Korea', 'facility': 'CHA Bundang Medical Center', 'geoPoint': {'lat': 37.43861, 'lon': 127.13778}}], 'overallOfficials': [{'name': 'Wonkyung Song', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'CHA Bundang Medical Center'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'CHABiotech CO., Ltd', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}