Fuchs Focus is a planned clinical study for Fuchs endothelial corneal dystrophy. This page provides patients, their families and care givers with information about the trial and trial participation. We plan to start the trial in the second quarter of 2021.

What is Fuchs Focus?

What is Fuchs Focus?

Fuchs Focus is the name of a clinical trial which aims to study whether the investigational RNA therapy QR-504a is safe for people with Fuchs endothelial corneal dystrophy (FECD) due to trinuclear repeat (TNR) expansion mutations in the TCF4 gene.

This Phase 1 trial is the first clinical study to investigate QR-504a. If you are interested in joining this trial, please read about the eligibility criteria.

Fuchs Focus logo
What is QR-504a?

What is QR-504a?

QR-504a is an investigational RNA therapy that aims to prevent or slow down vision loss in people with Fuchs endothelial corneal dystrophy (FECD) due to trinuclear repeat expansion mutations in the TCF4 gene.

The eyes of an elderly woman are examined by a female doctor
How is the trial set up?

How is the Fuchs Focus trial set up?

Clinical trials are used by researchers to find out whether new medicines are effective and safe. Key aspects of the Fuchs Focus trial are the following:

  • Fuchs Focus aims to find out whether QR-504a is safe and well-tolerated, and whether it has an effect on FECD biomarkers;
  • The study will enroll approximately 10 participants that are scheduled to receive corneal transplant (DMEK) and lens replacement surgery in both eyes due to advanced stage disease;
  • Study participants will receive a single intravitreal injection (injection into the eye) with QR-504a.

Participants will receive the study medicine once in the chosen (study) eye, which is the last eye to receive the corneal transplant.

Is Fuchs Focus for me?

Is Fuchs Focus for me?

The information below outlines the main criteria for participation in the trial. Participants must:

  • Have an established genetic diagnosis of FECD caused by trinuclear repeat (TNR) expansion mutations in the TCF4 gene;
  • Be scheduled to receive corneal transplant (DMEK) and lens replacement surgery in both eyes;
  • Not participate in another clinical study during the Fuchs Focus trial.
How to participate?

How to participate in the clinical trial?

We recommend that you discuss your situation and suitability for the trial with your doctor. They will be able to refer you to a medical center where the Fuchs Focus trial is conducted. You can also contact the trial center directly.

Fuchs Focus will soon open at the following location: Moorfields Eye Hospital in London, UK.

What is FECD?

What is Fuchs endothelial corneal dystrophy?

Fuchs endothelial corneal dystrophy (FECD) is a common genetic disease that affects the cornea, the most outer layer of the eye. FECD leads to progressive degeneration of a thin layer of cells just underneath the cornea, the corneal endothelium. When this layer is not functioning well it can result in corneal edema, scarring, corneal clouding, and vision loss. Blisters on the cornea are a major cause of pain in patients with advanced FECD.

FECD is a common disorder, studies show that it affects more than 4% of people over the age of 40 in the United States, and similar numbers are reported for other parts of the World. FECD is often caused by trinuclear repeat (TNR) expansion mutations in the TCF4 gene. In people of European descent, around 75% of FECD patients have this mutation.

Currently there are no treatment options available to stop or slow down FECD and disease management is aimed to reduce symptoms. The only effective therapy for late-stage FECD is corneal transplantation. The availability of donors, risk of rejection, and the inherent risk of such surgeries are some of the limitations of this option.

FAQ

Frequently asked questions

Below you will find answers to the most frequent questions about the Fuchs Focus trial.

  • Intravitreal injection is one of the most commonly performed procedures for eye diseases. The inside of the eye is filled with a jelly-like fluid (vitreous). During an intravitreal injection, the eye doctor will inject medicine into the vitreous with a very small needle, after numbing the eye. The patient may feel some pressure but no pain.

    Learn more about intravitreal injection

  • An RNA therapy is designed to correct the mistake, or mutation, in the RNA of someone with a genetic disease. By correcting the mistake, the RNA can then be used to create the protein that the cell needs, taking away the underlying cause of the disease.

    Learn more about how RNA therapy works

  • Yes, travel and accommodation costs will be covered.

  • While available information on QR-504a is encouraging, there is still much to learn about QR-504a and its effects. Therefore, QR-504a is currently not available outside clinical trials.

  • People participate in clinical trials for different reasons. Some participate because they want to learn more about their disease. Others participate because they want to help with the development of new treatments that could help others in the future.

    Clinical trials follow a specific set of standards and are strictly regulated to help keep all participants safe. All clinical trials are reviewed and approved by a committee of independent experts (Ethics Committee or Institutional Review Board).

    During a clinical trial, participants are carefully monitored. In addition to eye tests, general safety tests may be performed during study visits, such as blood tests, blood pressure, heart rate and temperature.

    Learn more about clinical trials

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