[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06275620":3,"trial-entities:NCT06275620":170,"trial-summary:NCT06275620":174},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":27,"primary_purpose":28,"phases":29,"enrollment_info":31,"interventions":34,"primary_outcomes":49,"secondary_outcomes":55,"sex":83,"minimum_age":84,"maximum_age":85,"healthy_volunteers":86,"eligibility_criteria":87,"std_ages":102,"locations":106,"central_contacts":162,"overall_officials":163,"references":164,"see_also_links":169},"NCT06275620","AGTC-RPGR-001 DAWN","A Study Comparing Two Doses of AGTC-501 in Male Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (DAWN)","A Phase 1\u002F2 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","ENROLLING_BY_INVITATION","2029-12","2024-10","2024-10-30","2023-11-14","Beacon Therapeutics","INDUSTRY",true,"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.","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.",[19],"X-Linked Retinitis Pigmentosa",[21,22,23,24,25,26],"XLRP","retinal degeneration","RPGR","adeno-associated virus","gene therapy","AAV","INTERVENTIONAL","TREATMENT",[30],"PHASE2",{"count":32,"type":33},24,"ESTIMATED",[35,41,45],{"type":36,"name":37,"description":38,"armGroupLabels":39},"BIOLOGICAL","AGTC-501 (high dose and standard corticosteroid regimen)","Adeno-associated virus vector expressing a human RPGR gene",[40],"Group 1 (High Dose, Standard Corticosteroid)",{"type":36,"name":42,"description":38,"armGroupLabels":43},"AGTC-501 (low dose and standard corticosteroid regimen)",[44],"Group 2 (Low Dose, Standard Corticosteroid)",{"type":36,"name":46,"description":38,"armGroupLabels":47},"AGTC-501 (high dose and modified corticosteroid regimen)",[48],"Group 3 (High Dose, Modified Corticosteroid)",[50,53],{"measure":51,"timeFrame":52},"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).","Day 0 - Month 12",{"measure":54,"timeFrame":52},"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).",[56,58,60,62,64,67,69,71,73,75,77,80],{"measure":57,"timeFrame":52},"The number of participants experiencing treatment-emergent AEs of ocular\u002Fnon-ocular adverse events, including treatment-emergent serious AEs.",{"measure":59,"timeFrame":52},"The proportion of participants experiencing treatment-emergent AEs of ocular\u002Fnon-ocular adverse events, including treatment-emergent serious AEs.",{"measure":61,"timeFrame":52},"Change from baseline in mean sensitivity across the whole grid, as measured by MAIA (Macular Integrity Assessment) microperimetry, assess photoreceptor function under low light",{"measure":63,"timeFrame":52},"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.",{"measure":65,"description":66,"timeFrame":52},"Change from baseline in full-field stimulus threshold (FST)","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":68,"timeFrame":52},"Change from baseline in Best Corrected Visual Acuity (BCVA) using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity",{"measure":70,"timeFrame":52},"Change from baseline in Low Luminance Visual Acuity (LLVA) using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity",{"measure":72,"timeFrame":52},"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)",{"measure":74,"timeFrame":52},"Change from baseline in ellipsoid zone (EZ) area measured by spectral domain optical coherence tomography (SD OCT)",{"measure":76,"timeFrame":52},"Change from baseline in seven domain scores from a Michigan Retinal Degeneration Questionnaire (MRDQ)",{"measure":78,"description":79,"timeFrame":52},"Change from baseline in Ora-VNC (visual navigation course) mobility test score","As assessed by functional assessment Ora-VNC (visual navigation course) mobility course",{"measure":81,"description":82,"timeFrame":52},"Change from baseline in the MObility Standardized Test-Virtual Reality (MOST-VR) mobility course test score","As measured by the MObility Standardized Test-Virtual Reality (MOST-VR) mobility course","MALE","12 Years",null,false,{"inclusion":88,"exclusion":95,"raw_text":101},[89,90,91,92,93,94],"Be ≥12 years of age","Have one eye previously treated with an AAV vector-based gene therapy designed to provide full-length functioning RPGR protein.","Have a BCVA no better than 78 letters and no worse than 34 letters","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.","Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, as determined by the Investigator and confirmed by the Central Reading Center (CRC).","Have detectable EZ line in the study eye as assessed by SD-OCT and confirmed by the CRC.",[96,97,98,99,100],"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.","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","Had intraocular surgery within 90 days of study treatment administration.","Have any active ocular\u002Fintraocular infection or inflammation","Have a history of steroid-induced raised IOP of \\>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.","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\u002Fintraocular infection or inflammation\n* Have a history of steroid-induced raised IOP of \\>25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.",[103,104,105],"CHILD","ADULT","OLDER_ADULT",[107,116,123,131,139,146,154],{"facility":108,"city":109,"state":110,"zip":111,"country":112,"geoPoint":113},"University of Florida","Jacksonville","Florida","32209","United States",{"lat":114,"lon":115},30.33218,-81.65565,{"facility":117,"city":118,"state":110,"zip":119,"country":112,"geoPoint":120},"Bascom Palmer Eye Institute","Miami","33136",{"lat":121,"lon":122},25.77427,-80.19366,{"facility":124,"city":125,"state":126,"zip":127,"country":112,"geoPoint":128},"Boston Children's Hospital","Boston","Massachusetts","02115",{"lat":129,"lon":130},42.35843,-71.05977,{"facility":132,"city":133,"state":134,"zip":135,"country":112,"geoPoint":136},"Cincinnati Eye Institute","Cincinnati","Ohio","45242",{"lat":137,"lon":138},39.12711,-84.51439,{"facility":140,"city":141,"state":134,"zip":142,"country":112,"geoPoint":143},"Cleveland Clinic","Cleveland","44195",{"lat":144,"lon":145},41.4995,-81.69541,{"facility":147,"city":148,"state":149,"zip":150,"country":112,"geoPoint":151},"Casey Eye Institute","Portland","Oregon","97239",{"lat":152,"lon":153},45.52345,-122.67621,{"facility":155,"city":156,"state":157,"zip":158,"country":112,"geoPoint":159},"Retina Foundation of the Southwest","Dallas","Texas","75231",{"lat":160,"lon":161},32.78306,-96.80667,[],[],[165],{"pmid":166,"type":167,"citation":168},"40547876","DERIVED","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\u002Fjoph\u002F7014745. eCollection 2025.",[],{"nct_id":4,"conditions":171,"biomarkers":173},[172],"X-linked retinitis pigmentosa",[],{"nct_id":4,"found":86,"summary":85,"prompt_version":85}]