protocolSection identificationModule nctId: NCT06333249, orgStudyIdInfo id: AGTC-RPGR-001 SKYLINE, briefTitle: A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE), statusModule overallStatus: ACTIVE_NOT_RECRUITING, startDateStruct date: 2021-04-13, primaryCompletionDateStruct date: 2023-04-11, completionDateStruct date: 2027-02, studyFirstPostDateStruct date: 2024-03-27, lastUpdatePostDateStruct date: 2024-03-27, sponsorCollaboratorsModule leadSponsor name: Beacon Therapeutics, class: INDUSTRY, descriptionModule briefSummary: This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations., conditionsModule conditions: X-Linked Retinitis Pigmentosa, designModule studyType: INTERVENTIONAL, phases: PHASE2, designInfo allocation: RANDOMIZED, interventionModel: PARALLEL, interventionModelDescription: The purpose of the Phase 2 study is to evaluate the efficacy, safety, and tolerability of two doses of AGTC-501 in male subjects between 8 - 50 years of age (inclusive) with XLRP genetically confirmed by at least one pathogenic variant in the RPGR gene.Approximately 12 subjects who meet the inclusion criteria, will be randomized in a 1:1 ratio to 1 of 2 treatment groups. Each subject will receive the assigned dose of AGTC-501 in the study eye; no treatment will be administered in the fellow eye. As treatment outcomes in pediatric vs. adult subjects may differ, randomization to dose groups will be stratified by age.Each subject will receive a central subretinal injection of AGTC-501 at the assigned dose in the central macula of the study eye., primaryPurpose: TREATMENT, maskingInfo masking: QUADRUPLE, maskingDescription: The Phase 2 study is a masked study; therefore, neither the investigator nor the subject will know the dose assignment. Both the subject and the investigator will know which eye received treatment., whoMasked: PARTICIPANT, whoMasked: CARE_PROVIDER, whoMasked: INVESTIGATOR, whoMasked: OUTCOMES_ASSESSOR, enrollmentInfo count: 14, type: ACTUAL, armsInterventionsModule interventions name: rAAV2tYF-GRK1-RPGR, outcomesModule primaryOutcomes measure: The difference in the proportion of responding eyes between treated and control eyes in the low dose group and high dose group at 12 months, as measured by MAIA microperimetry, where response is defined as a 7dB or more improvement in at least 5 loci., secondaryOutcomes measure: Proportion of responding eyes in treated eyes versus control eyes in the low dose group and the high dose group at Month 12 where responder is defined as an ORA-VNC mobility test score improvement of 2 or more luminance levels., secondaryOutcomes measure: Proportion of responding eyes in treated versus control eyes in the low dose group and the high dose group at Month 12, as measured by MAIA microperimetry, where responder is defined as a 7 dB or more improvement in at least 5 loci within bleb., secondaryOutcomes measure: Proportion of responding eyes in treated vs control eyes in the low dose group and the high dose group at Month 12, measured by MAIA microperimetry where responder is defined as 7 dB or more improvement in at least 5 loci within the central 16 loci, secondaryOutcomes measure: Difference in mean change from baseline in the central 36 loci (C36) mean sensitivity, as measured by MAIA microperimetry, in treated eyes versus control eyes in the low dose group and high dose group at Month 12., secondaryOutcomes measure: Difference in mean change from baseline in "within bleb" mean sensitivity, as measured by MAIA microperimetry, in treated eyes versus control eyes in the low dose group and high dose group at Month 12., secondaryOutcomes measure: Difference in mean change from baseline in central 10 degrees of vision on light adapted static perimetry, as measured by Octopus 900, in treated eyes versus control eyes in the low dose group and high dose group at Month 12., secondaryOutcomes measure: Difference in mean change from baseline in BCVA, as measured by ETDRS or tumbling "E" chart, in treated eyes versus control eyes in the low dose group and high dose group at Month 12., secondaryOutcomes measure: Proportion of responding eyes in treated versus control eyes in the low dose group and high dose group at Month 12 where responder is defined as a 10-letter vision gain as measured by ETDRS or tumbling "E" chart., secondaryOutcomes measure: Difference in mean change from baseline in the EZ area, as measured by SD-OCT, in treated eyes versus control eyes in the low dose group and high dose group at Month 12 Visit., secondaryOutcomes measure: Mean change from baseline in Impact of Vision Impairment (IVI) (Weih et al, 2002; Lamoureaux et al, 2007) or Impact of Vision Impairment for Children (IVI-C) (Cochrane et al, 2008) in the low dose group and high dose group at Month 12 Visit, secondaryOutcomes measure: Mean change from baseline in Patient Global Impressions of Change (PGI-C) and Patient Global Impressions of Severity (PGIS) in the low dose group and high dose group at Month 12 Visit., eligibilityModule sex: MALE, minimumAge: 8 Years, maximumAge: 50 Years, stdAges: CHILD, stdAges: ADULT, contactsLocationsModule locations facility: University of Florida, city: Jacksonville, state: Florida, zip: 32209, country: United States, geoPoint lat: 30.33218, lon: -81.65565, locations facility: Boston Children's Hosptial, city: Boston, state: Massachusetts, zip: 02115, country: United States, geoPoint lat: 42.35843, lon: -71.05977, locations facility: Cincinnati Eye Institute, city: Cincinnati, state: Ohio, zip: 45242, country: United States, geoPoint lat: 39.12713, lon: -84.51435, locations facility: Cleveland Clinic, city: Cleveland, state: Ohio, zip: 44195, country: United States, geoPoint lat: 41.4995, lon: -81.69541, locations facility: Casey Eye Institute, city: Portland, state: Oregon, zip: 97239, country: United States, geoPoint lat: 45.52345, lon: -122.67621, locations facility: Retina Foundation of the Southwest, city: Dallas, state: Texas, zip: 75231, country: United States, geoPoint lat: 32.78306, lon: -96.80667, hasResults: False