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
Sham procedure: Sham procedures (i.e. no penetration of the globe) closely mimic the active injection and serve to mask subjects to treatment assignment
Study summary
This study evaluates the safety, tolerability and efficacy of QR-1123 injection in the eye (intravitreal; IVT) injections (one eye/unilateral) in subjects receiving a single dose or repeat doses. Single injections will be assessed in an open label way, and repeat injections will be assessed in a double-masked, randomized, sham-controlled fashion.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Main Inclusion Criteria:
1. Male or female, ≥ 18 years of age.
2. Clinical presentation consistent with adRP, based on ophthalmic examinations.
3. Impairment on VF in the opinion of the Investigator, as determined by perimetry.
4. A molecular diagnosis of autosomal dominant form of RP with the P23H mutation in the RHO gene, based on genetic analysis.
5. A clear ocular media and adequate pupillary dilation to permit good quality fundus imaging, as assessed by the Investigator.
Main Exclusion Criteria:
1. 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).
2. 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.
Primary outcome measure(s)
Incidence and Severity of ocular AEs — up to 12 months Incidence and severity of ocular adverse events scored based on CTCAC in the study and fellow eye
Incidence and Severity of non-ocular AEs — up to 12 months Incidence and severity of non-ocular adverse events scored based on CTCAC in the study and fellow eye
Trial sites (5)
Facility
City
Region
Status
Sue Anschutz-Rogers Eye Center, University of Colorado - Dept. of Ophthalmology
Aurora
Colorado
VitreoRetinal Associates
Gainesville
Florida
Shriners UK Ophthalmology - University of Kentucky
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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