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WithdrawnNCT05052554Fuchs FocusUpdated May 11, 2022

Study With QR-504a to Evaluate Safety, Tolerability & Corneal Endothelium Molecular Biomarker(s) in Subjects With FECD3

A Phase 1 interventional study of QR-504a in Fuchs Endothelial Corneal Dystrophy, FECD3 and Corneal Diseases, sponsored by ProQR Therapeutics. Withdrawn at 1 site in United Kingdom. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-05-11.

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

Why this study was withdrawn
No patients
Phase
Phase 1
Study type
Interventional
Enrollment
0
Allocation
Non-randomized
Ages
18 Years and older
Sex
All
01

Study summary

PQ-504a-001 (Fuchs Focus) is an open-label, single-dose, exploratory study to evaluate safety, tolerability, and corneal endothelium molecular biomarker(s) in subjects with Fuchs Endothelial Corneal Dystrophy with Trinucleotide Repeat Expansion in the TCF4 gene (FECD3).

Read the detailed description

PQ-504a-001 (Fuchs Focus) is an open-label, intra-subject-controlled study in subjects with FECD3 scheduled for Descemet's Membrane Endothelial Keratoplasty (DMEK) in both eyes or with concurrent, preemptive lens replacement surgery (LRS/DMEK), if indicated as part of routine clinical practice. Each subject will go through the following two parts in this study:

  • Part A without study drug administration: Part A of the study starts once LRS/DMEK of the first eye (hereafter called Eye 1) has been scheduled and subject eligibility has been confirmed. During the LRS/DMEK of Eye 1 corneal endothelium will be collected to assess the levels of selected molecular biomarkers. According to routine clinical practice, subjects will then be monitored for at least 4 weeks and up to approximately 6 months to evaluate recovery of Eye 1 prior to scheduling LRS/DMEK of the second eye (hereafter called Eye 2). The duration of the follow-up period also depends on the availability of cornea transplant material for Eye 2. Data generated during this part will serve as intra-subject control for data generated in Part B.
  • Part B with study drug administration: Part B of the study starts once LRS/DMEK in Eye 2 can be scheduled and subject eligibility for Part B has been confirmed. At least 4 weeks prior to the scheduled LRS/DMEK in Eye 2, the subject will receive a single dose of QR-504a administered intravitreally in Eye 2. During the LRS/DMEK in Eye 2 corneal endothelium will be collected for molecular biomarker assessments and subjects will continue to be followed for safety and tolerability for at least 12 months post-surgery (ie, at least 13 months post dosing).
02

Conditions studied

  • Fuchs Endothelial Corneal Dystrophy
  • FECD3
  • Corneal Diseases
  • LRS
  • Descemet's Membrane Disorder
03

In context

Fuchs' Endothelial Dystrophy

92 studies on the registry are indexed under Fuchs' Endothelial Dystrophy; 28 are open to participants now.

Browse Fuchs' Endothelial Dystrophy 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

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Clinical diagnosis of FECD3 with:
  • confirmed presence of trinucleotide repeat (TNR) expansion in the TCF4 gene (50 TNRs or more as determined by PCR),
  • symmetrical disease progression in both eyes, in the opinion of the Investigator,
  • a clinical indication for DMEK in both eyes ((phakic, aphakic, pseudophakic), or a clinical indication for concurrent, preemptive lens replacement surgery \& DMEK in phakic eye.

Exclusion criteria

Exclusion Criteria:

  • Presence of any significant ocular or non-ocular disease/disorder which 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.
  • History of any form of keratoplasty in either eye.
  • History or presence of ocular herpetic diseases (including herpes simplex virus, varicella zoster or cytomegalovirus) in either eye.
  • Presence of any active ocular infection in either eye.
  • Receipt within 3 months prior to Screening of in the opinion of the investigator any procedures or drugs that cause ocular disease or have ocular side effects that may confound assessment of safety, local tolerability or potential efficacy including.
  • Systemic therapies known to influence the immune system (including but not limited to cytostatics, interferons, TNF-binding proteins, drugs acting on immunophilins, or antibodies with known impact on the immune system). Subjects that have been treated with systemic steroids within the past 12 months or that require intermittent use of topical steroids (eg, in pulmonological, dermatological or ophthalmological indications) may be considered for inclusion following approval by the Medical Monitor.
  • Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the study period.
  • Any prior receipt of genetic or stem-cell therapy for ocular or non-ocular disease.
  • Ongoing event of intraocular tumors.
  • Pregnant or breastfeeding subjects. Female subjects of childbearing potential and male subjects must be sexually inactive by abstinence, which is consistent with the preferred and usual lifestyle of the subject, or agree to use highly effective methods of birth control, as defined in the study protocol. Women of non-childbearing potential may be included without the use of adequate birth control, provided the participant meets the criteria.
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    30 µg cohort

    Open label single dose cohort: Dose level 1

    Drug: QR-504a

  • Experimental
    60 µg cohort

    Open label single dose cohort: Dose level 2

    Drug: QR-504a

Interventions

  • DrugQR-504a

    Intravitreal (IVT) injection

06

What researchers measure

Primary outcomes

  1. Incidence and severity of ocular and non-ocular adverse events (AEs)

    Time frame: 20 months

  2. Change from baseline in the isoform levels of MBNL1 and MBNL2 in corneal endothelium

    Time frame: 20 months

07

Study locations

1 site
  • Moorfields Eye Hospital, NIHR Clinical Research Facility
    London, United Kingdom
08

References and documents

Related links

Individual participant data

Plan to share: No

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Aug 2021 to primary completion in Apr 2022, 8 months.

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

Registered
Aug 6, 2021
First posted
Sep 22, 2021
StartActual
Aug 10, 2021
Primary completionActual
Apr 12, 2022
CompletionActual
Apr 12, 2022
Last update
May 11, 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 11, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05052554
Lead sponsor
ProQR Therapeutics
Responsible party
Sponsor
First posted
Sep 22, 2021
Start date
Aug 10, 2021
Primary completion
Apr 12, 2022
Completion
Apr 12, 2022
Last update
May 11, 2022

Study contacts

ProQR Medical Monitor
study director · ProQR Therapeutics

Oversight

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

Not currently enrolling

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

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