Status:
ACTIVE_NOT_RECRUITING
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
Lead Sponsor:
ProQR Therapeutics
Conditions:
Autosomal Dominant Retinitis Pigmentosa
Eye Diseases
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief 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. Sing...
Detailed Description
QR-1123 is an antisense oligonucleotide, designed to specifically target the mutant P23H messenger ribonucleic acid (mRNA) in order to reduce the expression of the P23H protein selectively, while pres...
Eligibility Criteria
Inclusion
- Main
- 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
- 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.
Key Trial Info
Start Date :
October 7 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 7 2022
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT04123626
Start Date
October 7 2019
End Date
June 7 2022
Last Update
May 6 2022
Active Locations (5)
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1
Sue Anschutz-Rogers Eye Center, University of Colorado - Dept. of Ophthalmology
Aurora, Colorado, United States, 80045
2
VitreoRetinal Associates
Gainesville, Florida, United States, 32607
3
Shriners UK Ophthalmology - University of Kentucky
Lexington, Kentucky, United States, 40536
4
Casey Eye Institute, OHSU
Portland, Oregon, United States, 97239