Clinical Trials Logo
  1. Home
  2. Eye Diseases
  3. NCT04123626

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  

Eye Diseases Clinical Trial

Official title:
A Prospective First-In-Human Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) Due to the P23H Mutation in the RHO Gene

Clinical Trial 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.

Clinical Trial 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 preserving expression of the wild type (WT) rhodopsin (RHO) protein. It is hypothesized that the reduction of mutant P23H mRNA will reduce the deleterious effects of the dominant-negative protein and should result in increased function of WT rhodopsin protein in photoreceptors. Restoration of WT RHO function is expected to improve vision in patients with adRP due to the P23H mutation. The study will comprise up to 8 single dose and repeat dose cohorts. Prior to initiating a higher single dose cohort and/or prior to initiating repeat dose cohort(s), available safety and efficacy data will be reviewed by the DMC. In the single dose cohorts subjects will receive a single, unilateral IVT injection of QR-1123 in an open label fashion. In the repeat dose cohorts subjects will be randomized to receive either a unilateral IVT injection of QR-1123 every 3 months or a unilateral sham procedure every 3 months, in a double masked fashion. Subjects will be followed for safety, tolerability and efficacy for a total period of 12 months. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT04123626
Study type Interventional
Source ProQR Therapeutics
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date October 7, 2019
Completion date June 7, 2022
View Details
See also
  Status Clinical Trial Phase
Recruiting NCT05414994 - Assessment of the Ocular Microbiome in Health and Disease
Active, not recruiting NCT04271709 - Manhattan Vision Screening and Follow-Up Study (NYC-SIGHT) N/A
Completed NCT02811692 - Study for Collection of Aflibercept Data in Routine Practice
Recruiting NCT06045299 - Evaluation of the Efficacy and Safety of LNZ101 and LNZ100 for the Presbyopia Phase 3
Recruiting NCT01521065 - An Open-label Study to Evaluate the Clinical and Economic Benefits of I-Ray in Patients With Choroidal Neovascularization Secondary to Age-related Macular Degeneration Phase 2
Completed NCT04799704 - Tear Film SARS-nCoV-2 Detection in Symptomatic and Pauci-symptomatic Patients.
Recruiting NCT05876689 - Swept Source OCT Imaging With the DREAM VG-OCT
Recruiting NCT04150432 - Correlation Between Exhaled Propofol Concentration With Plasma Concentration in Children N/A
Not yet recruiting NCT05550740 - Repeated Low-Level Red-Light Therapy for Shortening Axial Length N/A
Completed NCT02332343 - Sparing of the Fovea in Geographic Atrophy Progression N/A
Completed NCT01727973 - Efficacy of Subantimicrobial Dose Doxycycline for Moderate to Severe and Active Graves' Orbitopathy Phase 1/Phase 2
Terminated NCT01225146 - Study of 2.0 mg Ranibizumab in Subjects With Ischemic Central Retinal Vein Occlusion (RAVE2) Phase 1
Completed NCT00333203 - Next Generation Ophthalmic Irrigating Solution Posterior Segment Study Phase 3
Not yet recruiting NCT05565547 - Multimodal Equipment for Teleophthalmology Assessment (META)
Recruiting NCT05158699 - Effectiveness of Periocular Drug Injection in CATaract Surgery Phase 3
Recruiting NCT06451172 - Novel Antisense Oligonucleotide Eye Drops for Treating Antibiotic-Resistant Bacterial Keratitis Early Phase 1
Recruiting NCT06112340 - A Phase 2b Extension Study of Two Doses of Linsitinib in Subjects With Active, Moderate to Severe Thyroid Eye Disease (TED) Phase 2/Phase 3
Completed NCT05211089 - Manual Versus Automated Choroidal Thickness Measurements Using Swept-source Anterior Segment OCT
Not yet recruiting NCT06070467 - Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases
Completed NCT02946879 - Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)