Aurora is a clinical study for retinitis pigmentosa. This page provides patients, their families and care givers with information about the trial.
Aurora - Phase 1/2 clinical trial for RHO mediated retinitis pigmentosa
What is Aurora?
Aurora is the name of a clinical trial which aims to study whether the investigational RNA therapy QR-1123 is effective and safe for people with retinitis pigmentosa (RP) due to the P23H mutation, also known as c.68C>A, in the rhodopsin (RHO) gene.
Mutations in the RHO gene cause an autosomal dominant form of RP (adRP) and the P23H mutation is the most common mutation causing adRP in the United States.
This Phase 1/2 trial was launched in December 2019 and is the first clinical study to investigate QR-1123.
This trial is not enrolling participants at this time. Please sign up for our newsletter if you want to stay informed on the progress in development and to learn more about other opportunities for trial participation.
More information about the trial is also available on worldwide clinical trial database clinicaltrials.gov with identifier (NCT number): NCT04123626.
What is QR-1123?
QR-1123 is an investigational RNA therapy that aims to stop vision loss or restore vision in people that have RP due to the P23H mutation in the RHO gene.
How is the Aurora trial set up?
Clinical trials are used by researchers to find out whether new medicines are effective and safe. Key aspects of the Aurora trial are the following:
- Aurora aims to find out whether QR-1123 is safe and well-tolerated, and whether it is effective at stabilizing or improving vision;
- The study enrolled eleven adults (age 18 and up);
- Study participants received a single intravitreal injection (injection into the eye) with QR-1123 at one of five different doses.
Based on the results from Aurora, we plan to start a Phase 2 repeated dose clinical trial.
Genetic testing is the only way to receive an accurate diagnosis and will help find out if there are treatments or clinical trials available.Genetic testing
What is RHO mediated retinitis pigmentosa?
Retinitis pigmentosa (RP) is an inherited retinal disease that leads to vision loss and blindness. Symptoms usually start with night blindness during adolescence or early adulthood and further progress with loss of side (peripheral) vision leading to tunnel vision. Loss of central vision appears which frequently progresses to blindness in adulthood.
RP is a genetic disease that causes a mistake, or mutation, in the patient’s RNA. Because of the mutation, an essential protein in the eye cannot function, and this leads to the deterioration of the light detecting cells in the retina. RP can be caused by mutations in many different genes. In the United States, people often have RP because of the P23H mutation in the RHO gene causing a version of RP called autosomal dominant RP. Because of the mutation the RHO RNA produces a mutated version of the rhodopsin protein that is toxic to the retina.
In the United States, approximately 2,500 people have vision loss due to the P23H mutation. To date, there are no other therapies approved or in clinical development that treat the vision loss associated with this mutation.
Frequently asked questions
Below you will find answers to the most frequent questions about the Aurora 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.
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.
While available information on QR-1123 is encouraging, there is still much to learn about QR-1123 and its effects. Therefore, QR-1123 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 them and others in the future.
If you take part in a clinical trial, you may be one of the first people to benefit from new study medication. 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.
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