Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
X-Linked Retinitis Pigmentosa
About this trial
This is an interventional treatment trial for X-Linked Retinitis Pigmentosa focused on measuring XLRP, retinal degeneration, RPGR, adeno-associated virus, gene therapy, AAV
Eligibility Criteria
Phase 1/2 Dose Escalation Inclusion Criteria:
- Male subjects between the ages of 6-50 years old with a documented RPGR mutation within exons 1-14 and/or ORF15 from a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory;
- Clinical diagnosis of X-linked retinitis pigmentosa (XLRP);
- Best-corrected visual acuity not better than 78 ETDRS letters (20/32) in the study eye;
- Also meet the other requirements of the study as specified in the protocol
Phase 1/2 Dose Escalation Exclusion Criteria:
• Have other known retinal disease mutations or previously received an AAV gene therapy product, as well as being unable or unwilling to meet the requirements of the study
Phase 2 Dose Expansion Inclusion Criteria:
- Males between the ages of 8-50 years old with a clinical diagnosis of XLRP with a confirmed RPGR mutation who also meet the other requirements of the study
- Have at least one documented pathogenic or likely pathogenic variant in the RPGR gene within exons 1-14 and/or ORF15 from Molecular Vision Laboratory (MVL), a CLIA-certified laboratory.
- Both eyes: Have a BCVA no better than 75 letters (20/32) and no worse than 35 letters (20/200) in both eyes based on an ETDRS chart at each screening visit. Pediatric subjects unable to read the ETDRS letters may utilize a tumbling "E" chart for BCVA assessments.
Phase 2 Dose Expansion Exclusion Criteria
• Have other known retinal disease mutations or previously received an AAV gene therapy product, as well as being unable or unwilling to meet the requirements of the study
Sites / Locations
- University of FloridaRecruiting
- Boston Children's HospitalRecruiting
- Columbia University
- Duke Eye Center
- Cincinnati Eye InstituteRecruiting
- Casey Eye Institute, Oregon Health and Science UniversityRecruiting
- Retina Foundation of the SouthwestRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Experimental
Experimental
Experimental
Experimental
Experimental
Group 1: Phase 1/2 Dose Escalation
Group 2: Phase 1/2 Dose Escalation and Low Dose Group Phase 2 Dose Expansion
Group 3 and Group 4 Phase 1/2 Dose Escalation
Group 5 Phase 1/2 Dose Escalation and High Dose Group Phase 2 Dose Expansion
Group 6 Phase 1/2 Dose Escalation
Male subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug.
Phase 1/2 Dose Escalation: male subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug. Phase 2 Dose Expansion: male subjects at least 8 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug. This will be the low dose group for the phase 2 expansion.
Group 3 male subjects at least 18 y/o and Group 4 male subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
Phase 1/2 Dose Escalation: male subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug. Phase 2 Dose Expansion: male subjects at least 8 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug. This will be the high dose group for the phase 2 expansion.
Male subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug.