Ignite Creation Date:
2025-12-25 @ 4:22 AM
Ignite Modification Date:
2025-12-26 @ 3:25 AM
Study NCT ID:
NCT06275620
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2024-10-30
First Post:
2024-02-16
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D012162', 'term': 'Retinal Degeneration'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D012164', 'term': 'Retinal Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 24}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2023-11-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-10', 'completionDateStruct': {'date': '2029-12', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-10-28', 'studyFirstSubmitDate': '2024-02-16', 'studyFirstSubmitQcDate': '2024-02-16', 'lastUpdatePostDateStruct': {'date': '2024-10-30', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-02-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-11', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The primary safety outcome is the number of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs).', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'The primary safety outcome is the proportion of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs).', 'timeFrame': 'Day 0 - Month 12'}], 'secondaryOutcomes': [{'measure': 'The number of participants experiencing treatment-emergent AEs of ocular/non-ocular adverse events, including treatment-emergent serious AEs.', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'The proportion of participants experiencing treatment-emergent AEs of ocular/non-ocular adverse events, including treatment-emergent serious AEs.', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Change from baseline in mean sensitivity across the whole grid, as measured by MAIA (Macular Integrity Assessment) microperimetry, assess photoreceptor function under low light', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Response, as measured by MAIA (Macular Integrity Assessment) microperimetry, where response is defined as a greater than or equal to 7 decibel (dB) visual sensitivity improvement from baseline in at least 5 loci.', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Change from baseline in full-field stimulus threshold (FST)', 'timeFrame': 'Day 0 - Month 12', 'description': 'As assessed by full-field stimulus threshold (FST); FST measures the sensitivity of the visual field by testing for the lowest luminance flash which elicits a visual sensation perceived'}, {'measure': 'Change from baseline in Best Corrected Visual Acuity (BCVA) using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Change from baseline in Low Luminance Visual Acuity (LLVA) using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Proportion of responding eyes in treated versus control eyes at Month 12 where responder is defined as an improvement of at least 15-letters on low-luminance visual acuity (LLVA)', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Change from baseline in ellipsoid zone (EZ) area measured by spectral domain optical coherence tomography (SD OCT)', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Change from baseline in seven domain scores from a Michigan Retinal Degeneration Questionnaire (MRDQ)', 'timeFrame': 'Day 0 - Month 12'}, {'measure': 'Change from baseline in Ora-VNC (visual navigation course) mobility test score', 'timeFrame': 'Day 0 - Month 12', 'description': 'As assessed by functional assessment Ora-VNC (visual navigation course) mobility course'}, {'measure': 'Change from baseline in the MObility Standardized Test-Virtual Reality (MOST-VR) mobility course test score', 'timeFrame': 'Day 0 - Month 12', 'description': 'As measured by the MObility Standardized Test-Virtual Reality (MOST-VR) mobility course'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['XLRP', 'retinal degeneration', 'RPGR', 'adeno-associated virus', 'gene therapy', 'AAV'], 'conditions': ['X-Linked Retinitis Pigmentosa']}, 'referencesModule': {'references': [{'pmid': '40547876', 'type': 'DERIVED', 'citation': 'Wang CY, Chen L, Lin TY, Huang SP. Systematic Identification of Candidate Genes for Inherited Retinal Disease Gene Therapy Integrating Worldwide IRD Cohort and Single-Cell Analysis. J Ophthalmol. 2025 Jun 12;2025:7014745. doi: 10.1155/joph/7014745. eCollection 2025.'}]}, 'descriptionModule': {'briefSummary': 'This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.', 'detailedDescription': 'This is a Phase 2, open-label, multicenter study to evaluate the safety of 2 doses of AGTC-501 administered as a single subretinal injection in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.\n\nThe trial includes a screening period of up to 60 days and a 5 year study period.\n\nEach participant will receive a single subretinal injection of one of two dose levels of AGTC-501 in their previously untreated eye. There will be 3 groups. Group 1 will receive the high dose and include up to 12 participants, Group 2 will receive the low dose and will include 6 participants, and Group 3 will include \\~3-6 participants. Participants in Groups 1 and 2 will receive the standard corticosteroid regimen. A single subretinal injection of the high dose AGTC-501 will be administered to participants in Group 1 (n = 12), while participants in Group 2 (n = 6) will receive a single subretinal injection of low dose AGTC-501. Group 2 (low dose AGTC-501, Standard Steroid) will be dosed before moving to Group 3. After 6 Group 1 (high dose) study participants reach post-operative Month 1, all data will be reviewed by the DSMC. If no safety signals arise, additional participants, Group 3 (n \\~ 3-6), will receive a single subretinal injection of the high dose with a modified course of corticosteroids.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'genderBased': True, 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Be ≥12 years of age\n* Have one eye previously treated with an AAV vector-based gene therapy designed to provide full-length functioning RPGR protein.\n* Have a BCVA no better than 78 letters and no worse than 34 letters\n* Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability and fixation, per the Investigator's discretion.\n* Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, as determined by the Investigator and confirmed by the Central Reading Center (CRC).\n* Have detectable EZ line in the study eye as assessed by SD-OCT and confirmed by the CRC.\n\nExclusion Criteria:\n\n* Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel that, in the opinion of the Investigator, would interfere with the potential therapeutic effect of the study agent or the quality of the assessments.\n* Have pre-existing eye conditions that would preclude the planned surgery, interfere with the interpretation of study endpoints, or increase the risk of surgical complications\n* Had intraocular surgery within 90 days of study treatment administration.\n* Have any active ocular/intraocular infection or inflammation\n* Have a history of steroid-induced raised IOP of \\>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy."}, 'identificationModule': {'nctId': 'NCT06275620', 'briefTitle': 'A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Beacon Therapeutics'}, 'officialTitle': 'A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa', 'orgStudyIdInfo': {'id': 'AGTC-RPGR-001 DAWN'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Group 1 (High Dose, Standard Corticosteroid)', 'description': 'Following a pars plana vitrectomy, the previously untreated eye of participants (n=up to 12) will receive a central subretinal injection at the high dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye.\n\nThe corticosteroid taper for all treated participants in Groups 1 and 2 (high and low doses with standard corticosteroid regimen) will be a standard taper over the course of several weeks.', 'interventionNames': ['Biological: AGTC-501 (high dose and standard corticosteroid regimen)']}, {'type': 'EXPERIMENTAL', 'label': 'Group 2 (Low Dose, Standard Corticosteroid)', 'description': 'Following a pars plana vitrectomy, the previously untreated eye of participants (n=6) will receive a central subretinal injection at the low dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye.\n\nThe corticosteroid taper for all treated participants in Groups 1 and 2 (high and low doses with standard corticosteroid regimen) will be a standard taper over the course of several weeks.', 'interventionNames': ['Biological: AGTC-501 (low dose and standard corticosteroid regimen)']}, {'type': 'EXPERIMENTAL', 'label': 'Group 3 (High Dose, Modified Corticosteroid)', 'description': 'Following a pars plana vitrectomy, the previously untreated eye of participants (n=approximately 3-6) will receive a central subretinal injection at the high dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye.\n\nParticipants in Group 3 (high dose, modified corticosteroid) will have a more rapid corticosteroid taper.', 'interventionNames': ['Biological: AGTC-501 (high dose and modified corticosteroid regimen)']}], 'interventions': [{'name': 'AGTC-501 (high dose and standard corticosteroid regimen)', 'type': 'BIOLOGICAL', 'description': 'Adeno-associated virus vector expressing a human RPGR gene', 'armGroupLabels': ['Group 1 (High Dose, Standard Corticosteroid)']}, {'name': 'AGTC-501 (low dose and standard corticosteroid regimen)', 'type': 'BIOLOGICAL', 'description': 'Adeno-associated virus vector expressing a human RPGR gene', 'armGroupLabels': ['Group 2 (Low Dose, Standard Corticosteroid)']}, {'name': 'AGTC-501 (high dose and modified corticosteroid regimen)', 'type': 'BIOLOGICAL', 'description': 'Adeno-associated virus vector expressing a human RPGR gene', 'armGroupLabels': ['Group 3 (High Dose, Modified Corticosteroid)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32209', 'city': 'Jacksonville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'Bascom Palmer Eye Institute', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '45242', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cincinnati Eye Institute', 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '44195', 'city': 'Cleveland', 'state': 'Ohio', 'country': 'United States', 'facility': 'Cleveland Clinic', 'geoPoint': {'lat': 41.4995, 'lon': -81.69541}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Casey Eye Institute', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '75231', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Retina Foundation of the Southwest', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Beacon Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}