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Clinical Trials in the USA / NCT04123626
Active, not recruiting Phase 1/2

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

NCT04123626 · tracked via the Priya Life Science USA tracker
Sponsor
ProQR Therapeutics
Phase
Phase 1/2
Started
2019-10-07
Last updated
2022-05-06

Condition(s) studied

Autosomal Dominant Retinitis PigmentosaEye DiseasesEye Diseases, HereditaryRetinal DystrophiesRetinal DiseaseRetinitisVision TunnelVision Disorders

Investigational drug(s) / intervention(s)

QR-1123Sham procedure

QR-1123: unilateral IVT injection

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)

Trial sites (5)

FacilityCityRegionStatus
Sue Anschutz-Rogers Eye Center, University of Colorado - Dept. of Ophthalmology Aurora Colorado
VitreoRetinal Associates Gainesville Florida
Shriners UK Ophthalmology - University of Kentucky Lexington Kentucky
Casey Eye Institute, OHSU Portland Oregon
Retina Foundation of the Southwest Dallas Texas

More ProQR Therapeutics trials in the USA

Other trials for the same condition

Official registry record

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.

View NCT04123626 on ClinicalTrials.gov ↗ ← All trials in the USA