Print

Purpose

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.

Conditions

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Outcomes Assessor)
Masking Description
Single dose cohorts are open label. Repeat dose cohorts are randomized, double masked.

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
QR-1123 Single dose - dose level 1 		Open label Single dose cohort: dose level 1
  • Drug: QR-1123
    unilateral IVT injection
Experimental
QR-1123 Single dose - dose level 2 		Open label Single dose cohort: dose level
  • Drug: QR-1123
    unilateral IVT injection
Experimental
QR-1123 Single dose - dose level 3 		Open label Single dose cohort: dose level 3
  • Drug: QR-1123
    unilateral IVT injection
Experimental
QR-1123 Single dose - dose level 4 		Open label Single dose cohort: dose level 4
  • Drug: QR-1123
    unilateral IVT injection
Experimental
QR-1123 Single dose - dose level 5 		Open label Single dose cohort: dose level 5
  • Drug: QR-1123
    unilateral IVT injection
Experimental
Repeat dose cohort 1 		Double-masked, randomized, sham controlled, Repeat dose cohort. Dose levels will be determined following DMC review of obtained safety and efficacy data.
  • Drug: QR-1123
    unilateral IVT injection
  • Other: Sham procedure
    Sham procedures (i.e. no penetration of the globe) closely mimic the active injection and serve to mask subjects to treatment assignment

Recruiting Locations

More Details

NCT ID
NCT04123626
Status
Active, not recruiting
Sponsor
ProQR Therapeutics

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 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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.