A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene (AURORA)
Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary
About this trial
This is an interventional treatment trial for Autosomal Dominant Retinitis Pigmentosa focused on measuring adRP, Retinitis Pigmentosa, P23H mutation, Rhodopsin, antisense oligonucleotide, IVT, autosomal dominant
Eligibility Criteria
Main Inclusion Criteria:
- Male or female, ≥ 18 years of age.
- Clinical presentation consistent with adRP, based on ophthalmic examinations.
- Impairment on VF in the opinion of the Investigator, as determined by perimetry.
- A molecular diagnosis of autosomal dominant form of RP with the P23H mutation in the RHO gene, based on genetic analysis.
- A clear ocular media and adequate pupillary dilation to permit good quality fundus imaging, as assessed by the Investigator.
Main Exclusion Criteria:
- Presence of additional pathogenic mutations in genes (other than the P23H mutation in the RHO gene) associated with inherited retinal degenerative diseases or syndromes, based on genetic analysis (eg, Usher syndrome, Leber congenital amaurosis, etc).
- Presence of any significant ocular or non-ocular disease/disorder (including medication and laboratory test abnormalities) which, in the opinion of the Investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or the subject's ability to participate in the study.
Sites / Locations
- Sue Anschutz-Rogers Eye Center, University of Colorado - Dept. of Ophthalmology
- VitreoRetinal Associates
- Shriners UK Ophthalmology - University of Kentucky
- Casey Eye Institute, OHSU
- Retina Foundation of the Southwest
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
QR-1123 Single dose - dose level 1
QR-1123 Single dose - dose level 2
QR-1123 Single dose - dose level 3
QR-1123 Single dose - dose level 4
QR-1123 Single dose - dose level 5
Repeat dose cohort 1
Open label Single dose cohort: dose level 1
Open label Single dose cohort: dose level
Open label Single dose cohort: dose level 3
Open label Single dose cohort: dose level 4
Open label Single dose cohort: dose level 5
Double-masked, randomized, sham controlled, Repeat dose cohort. Dose levels will be determined following DMC review of obtained safety and efficacy data.