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Active, not recruitingNCT04123626AURORAUpdated May 6, 2022

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

A Phase 1/2 interventional study of QR-1123 and Sham procedure in Autosomal Dominant Retinitis Pigmentosa, Eye Diseases and Eye Diseases, Hereditary, sponsored by ProQR Therapeutics. Active, not recruiting at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-05-06.

Sponsored by ProQR Therapeutics · Phase 1/2, Interventional, and Treatment

From the registry’s dates

  • Primary completion was expected by Jun 2022, 4 years 5 months ago, but the record still lists the study as active, not recruiting.
Phase
Phase 1/2
Study type
Interventional
Enrollment
11
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

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.

Read the 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.

02

Conditions studied

  • Autosomal Dominant Retinitis Pigmentosa
  • Eye Diseases
  • Eye Diseases, Hereditary
  • Retinal Dystrophies
  • Retinal Disease
  • Retinitis
  • Vision Tunnel
  • Vision Disorders

Keywords

  • adRP
  • Retinitis Pigmentosa
  • P23H mutation
  • Rhodopsin
  • antisense oligonucleotide
  • IVT
  • autosomal dominant
03

In context

Eye Diseases

388 studies on the registry are indexed under Eye Diseases; 62 are open to participants now.

This study's enrollment of 11 is below the median of 62 across 269 interventional studies indexed under Eye Diseases.

Browse Eye Diseases studies →

Lead sponsor

ProQR Therapeutics is the lead sponsor of 7 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

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.
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Sequential assignment
Masking
Triple (Participant, Care provider, Outcomes assessor)
Enrollment
11 participants (actual)

Study arms

  • Experimental
    QR-1123 Single dose - dose level 1

    Open label Single dose cohort: dose level 1

    Drug: QR-1123

  • Experimental
    QR-1123 Single dose - dose level 2

    Open label Single dose cohort: dose level

    Drug: QR-1123

  • Experimental
    QR-1123 Single dose - dose level 3

    Open label Single dose cohort: dose level 3

    Drug: QR-1123

  • Experimental
    QR-1123 Single dose - dose level 4

    Open label Single dose cohort: dose level 4

    Drug: QR-1123

  • Experimental
    QR-1123 Single dose - dose level 5

    Open label Single dose cohort: dose level 5

    Drug: QR-1123

  • 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 · Other: Sham procedure

Interventions

  • DrugQR-1123

    unilateral IVT injection

  • OtherSham procedure

    Sham procedures (i.e. no penetration of the globe) closely mimic the active injection and serve to mask subjects to treatment assignment

06

What researchers measure

Primary outcomes

  1. Incidence and Severity of ocular AEs

    Incidence and severity of ocular adverse events scored based on CTCAC in the study and fellow eye

    Time frame: up to 12 months

  2. Incidence and Severity of non-ocular AEs

    Incidence and severity of non-ocular adverse events scored based on CTCAC in the study and fellow eye

    Time frame: up to 12 months

Secondary outcomes

  1. Changes in BCVA

    Changes in Best corrected visual acuity (BCVA)

    Time frame: up to 12 months

  2. Changes in LLVA

    Changes in Low-luminance visual acuity (LLVA)

    Time frame: up to 12 months

  3. Changes in DAC perimetry

    Changes in Dark adapted chromatic (DAC) perimetry

    Time frame: up to 12 months

  4. Changes in Static VF

    Changes in Static VF (Visual Field)

    Time frame: up to 12 months

  5. Changes in Microperimetry

    Changes in Microperimetry

    Time frame: up to 12 months

  6. Changes in SD-OCT

    Changes in Spectral Domain-Optical Coherence Tomography

    Time frame: up to 12 months

  7. Changes in FST

    Changes in Full-field Stimulus Threshold (FST)

    Time frame: up to 12 months

  8. Changes in Full-field ERG

    Changes in Full-field Electroretinogram (ERG)

    Time frame: up to 12 months

  9. Assessment of systemic exposure after treatment with QR-1123

    Serum levels of QR-1123

    Time frame: up to 12 months

07

Study locations

5 sites
08

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Oct 2019 (actual). Primary completion is estimated for Jun 2022, 32 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Oct 1, 2019
First posted
Oct 11, 2019
StartActual
Oct 7, 2019
Primary completionEst.
Jun 7, 2022
CompletionEst.
Jun 7, 2022
Last update
May 6, 2022
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on May 6, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04123626
Lead sponsor
ProQR Therapeutics
Responsible party
Sponsor
First posted
Oct 11, 2019
Start date
Oct 7, 2019
Primary completion
Jun 7, 2022 (estimated)
Completion
Jun 7, 2022 (estimated)
Last update
May 6, 2022

Study contacts

ProQR Medical Monitor
study director · ProQR Therapeutics
ProQR Clinical Trial Manager
study director · ProQR Therapeutics

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in May 2022. You cannot join it, but the record below documents what was studied.

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