Fuchs Focus - v1
Research type
Research Study
Full title
OPEN-LABEL, SINGLE-DOSE, EXPLORATORY STUDY WITH QR-504a 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)
IRAS ID
274748
Contact name
Stephen Tuft
Contact email
Eudract number
2019-004565-40
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Summary of Research
The primary research question is whether QR-504a is safe and what the effect is on molecular disease signs in the eye after a single dose in patients with type 3 Fuchs Endothelial Corneal Dystrophy (FECD3)?
ProQR Therapeutics is developing an investigational product (QR-504a) designed to specifically target the underlying cause of FECD3 and to prevent or slow down the degeneration of the cornea.
FECD3 is an inherited disease characterized by the degeneration of the cornea (the transparent layer forming the front of the eye). The disease results in corneal swelling (edema), corneal clouding and vision loss. The only treatment for FECD is corneal transplantation when the disease is advanced. There is a need for treatments for FECD.
The study, PQ-504a-001, will be open to patients who have a clinical diagnosis of FECD3 and are eligible to have corneal transplantation (DMEK) in both eyes. This study consists of 2 parts: Part A, where DMEK of the first eye is performed as per routine care and data are collected as intra-subject control, and Part B where QR-504a is administered into the other eye a few weeks before the surgery of that eye. During both DMEK surgeries, the removed eye tissue (corneal endothelium) will be taken to measure molecular disease signs (biomarkers). Participating patients will continue to be followed for safety and tolerability for at least 12 months.
Approximately 9 patients are planned to participate in the study. The study will be performed in a center with expertise in corneal disease. This study will help understand if QR-504 is safe and how it works in the cornea of patients with FECD3 (biomarkers). Patients participating in this study will not benefit from it. This study will provide important information to help the development of QR-504a.Summary of Results
As the study closed prior to successful patient recruitment, there are no results to be published
REC name
West of Scotland REC 1
REC reference
20/WS/0023
Date of REC Opinion
19 Mar 2020
REC opinion
Further Information Favourable Opinion