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

NCT05052554 | Withdrawn Phase 1

• 1 other identifier: PQ-504a-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

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

Outcome Measures

Primary Outcomes (2)

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

  20 months

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

  20 months

Study Arms (2)

30 µg cohort

EXPERIMENTAL

Open label single dose cohort: Dose level 1

Drug: QR-504a

60 µg cohort

EXPERIMENTAL

Open label single dose cohort: Dose level 2

Drug: QR-504a

Interventions

QR-504a DRUG

Intravitreal (IVT) injection

30 µg cohort; 60 µg cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• ProQR Therapeutics: lead

Study Sites (1)

Moorfields Eye Hospital, NIHR Clinical Research Facility

London, United Kingdom

Location

Related Links

• Sponsor website

MeSH Terms

Conditions

Fuchs' Endothelial Dystrophy; Corneal Diseases

Condition Hierarchy (Ancestors)

Corneal Dystrophies, Hereditary; Eye Diseases; Eye Diseases, Hereditary; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• ProQR Medical Monitor

  ProQR Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: QR-504a will be administered per intravitreal (IVT) injection, as a single dose. The starting dose is 30 µg. Guided by the dose (de-)escalation plan, doses in a range of below 30 µg and up to 60 µg may be evaluated following pre-specified criteria for opening additional dose cohorts.

Study Record Dates

• First Submitted: August 6, 2021
• First Posted: September 22, 2021
• Study Start: August 10, 2021
• Primary Completion: April 12, 2022
• Study Completion: April 12, 2022
• Last Updated: May 11, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)