Ignite Creation Date:
2025-12-25 @ 2:41 AM
Ignite Modification Date:
2025-12-29 @ 10:56 AM
Study NCT ID:
NCT04671433
Status:
COMPLETED
Last Update Posted:
2025-09-29
First Post:
2020-11-05
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['OUTCOMES_ASSESSOR'], 'maskingDescription': 'No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 97}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2020-12-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-09', 'dispFirstSubmitDate': '2025-09-25', 'completionDateStruct': {'date': '2024-09-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-25', 'studyFirstSubmitDate': '2020-11-05', 'studyFirstSubmitQcDate': '2020-12-15', 'dispFirstPostDateStruct': {'date': '2025-04-25', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-09-29', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2020-12-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-09-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze', 'timeFrame': 'From Baseline to 52 Weeks', 'description': 'Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed.'}, {'measure': 'Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed.'}, {'measure': 'Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Pointwise response in full visual field at Week 52 will be assessed.'}, {'measure': 'Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed.'}, {'measure': 'Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Pointwise response in the central 30 degrees visual field at Week 52 will be assessed.'}, {'measure': 'Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed.'}, {'measure': 'Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed.'}, {'measure': 'Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52.'}, {'measure': 'Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52.'}, {'measure': 'Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52.'}, {'measure': 'Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52.'}, {'measure': 'Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52', 'timeFrame': 'From Baseline to Week 52', 'description': 'Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52.'}, {'measure': 'Number of Participants with Ocular and Non-ocular Adverse Events', 'timeFrame': 'Day 1 - Week 52', 'description': 'Number of participants with ocular and non-ocular adverse events will be assessed.'}, {'measure': 'Number of Participants With Abnormalities in Laboratory Assessments', 'timeFrame': 'Day 1 - 52 Weeks', 'description': 'Number of participants with abnormalities in laboratory assessments will be assessed.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['X-Linked Retinitis Pigmentosa']}, 'descriptionModule': {'briefSummary': 'A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or female\n* 3 years of age or older\n* Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory\n\nExclusion Criteria:\n\n* Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration\n* Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule\n* Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery'}, 'identificationModule': {'nctId': 'NCT04671433', 'briefTitle': 'Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene', 'organization': {'class': 'INDUSTRY', 'fullName': 'Janssen Research & Development, LLC'}, 'officialTitle': 'Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene', 'orgStudyIdInfo': {'id': 'CR109258'}, 'secondaryIdInfos': [{'id': 'MGT-RPGR-021', 'type': 'OTHER', 'domain': 'Janssen Research & Development, LLC'}, {'id': '2020-002873-88', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental - Immediate Treatment', 'description': 'Intermediate dose.', 'interventionNames': ['Biological: Genetic: AAV5-hRKp.RPGR']}, {'type': 'OTHER', 'label': 'Deferred Treatment', 'description': 'Deferred Treatment', 'interventionNames': ['Biological: Genetic: AAV5-hRKp.RPGR']}, {'type': 'EXPERIMENTAL', 'label': 'Experimental Immediate Treatment', 'description': 'Low dose.', 'interventionNames': ['Biological: Genetic: AAV5-hRKp.RPGR']}], 'interventions': [{'name': 'Genetic: AAV5-hRKp.RPGR', 'type': 'BIOLOGICAL', 'otherNames': ['botaretigene sparoparvovec'], 'description': 'Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group', 'armGroupLabels': ['Experimental - Immediate Treatment', 'Experimental Immediate Treatment']}, {'name': 'Genetic: AAV5-hRKp.RPGR', 'type': 'BIOLOGICAL', 'description': 'No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study)', 'armGroupLabels': ['Deferred Treatment']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92093 0946', 'city': 'La Jolla', 'state': 'California', 'country': 'United States', 'facility': 'Shiley Eye Institute Jacobs Retina Center', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Childrens Hospital', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94303', 'city': 'Palo Alto', 'state': 'California', 'country': 'United States', 'facility': 'Stanford Health Care', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '32607', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'VitreoRetinal Associates, PA', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Massachusetts General Hospital - Center for Celiac Research and Treatment', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48105', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'Univ of Michigan Medical Center', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '27705', 'city': 'Durham', 'state': 'North Carolina', 'country': 'United States', 'facility': 'Duke Eye Center', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'University of Pittsburgh Medical Center (UPMC)', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '9000', 'city': 'Ghent', 'country': 'Belgium', 'facility': 'UZ Gent', 'geoPoint': {'lat': 51.05, 'lon': 3.71667}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'Hospital For Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': '2600', 'city': 'Glostrup Municipality', 'country': 'Denmark', 'facility': 'Rigshospitalet Glostrup', 'geoPoint': {'lat': 55.6666, 'lon': 12.40377}}, {'zip': '75012', 'city': 'Paris', 'country': 'France', 'facility': "Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts", 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '91120', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Hadassah Medical Center', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'zip': '50134', 'city': 'Florence', 'country': 'Italy', 'facility': 'Azienda Ospedaliero Universitaria Careggi', 'geoPoint': {'lat': 43.77925, 'lon': 11.24626}}, {'zip': '20142', 'city': 'Milan', 'country': 'Italy', 'facility': 'Ospedale San Paolo', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '80131', 'city': 'Napoli', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli', 'geoPoint': {'lat': 40.87618, 'lon': 14.5195}}, {'zip': '00198', 'city': 'Roma', 'country': 'Italy', 'facility': 'IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '1105AZ', 'city': 'Amsterdam', 'country': 'Netherlands', 'facility': 'VUMC Amsterdam', 'geoPoint': {'lat': 52.37403, 'lon': 4.88969}}, {'zip': '6525EX', 'city': 'Nijmegen', 'country': 'Netherlands', 'facility': 'Radboudumc', 'geoPoint': {'lat': 51.8425, 'lon': 5.85278}}, {'zip': '28040', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hosp Univ Fund Jimenez Diaz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '4031', 'city': 'Basel', 'country': 'Switzerland', 'facility': 'University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical', 'geoPoint': {'lat': 47.55839, 'lon': 7.57327}}, {'zip': '1004', 'city': 'Lausanne', 'country': 'Switzerland', 'facility': 'Universite de Lausanne, Hopital ophtalmique Jules-Gonin', 'geoPoint': {'lat': 46.516, 'lon': 6.63282}}, {'zip': 'EH3 9HA', 'city': 'Edinburgh', 'country': 'United Kingdom', 'facility': 'NHS Lothian', 'geoPoint': {'lat': 55.95206, 'lon': -3.19648}}, {'zip': 'G12 0YN', 'city': 'Glasgow', 'country': 'United Kingdom', 'facility': 'Gartnavel General Hospital', 'geoPoint': {'lat': 55.86515, 'lon': -4.25763}}, {'zip': 'LS9 7TF', 'city': 'Leeds', 'country': 'United Kingdom', 'facility': 'St James University Hospital', 'geoPoint': {'lat': 53.79648, 'lon': -1.54785}}, {'zip': 'EC1V 2PD', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Moorfields Eye Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Janssen Research & Development, LLC Clinical Trial', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Janssen Research & Development, LLC'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Janssen Research & Development, LLC', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}