Aurora is a clinical study for retinitis pigmentosa. This page provides patients, their families and care givers with information about the trial and trial participation.
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. If you are interested in joining this trial, please read about the eligibility criteria.

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 will enroll adults (age 18 and up);
- Study participants will be randomly assigned to one of the following study groups:
- Single dose group: will receive one intravitreal injection (injection into the eye) with QR-1123.
- Multiple dose group: will receive up to four intravitreal injections with QR-1123 or up to four sham procedures (intravitreal injection is mimicked but no injection and no study medicine are given).
Participants will receive the study medicine or the sham procedure in the chosen (study) eye, which is the eye with the worse vision.
Is Aurora for me?
The information below outlines the main criteria for participation in the trial. Participants must:
- Have an established genetic diagnosis of RP caused by the P23H mutation (also known as c.68C>A) in the RHO gene;
- Be 18 years of age or older;
- Not participate in another clinical study during the Aurora trial.
Genetic testing
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
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 Aurora trial is conducted. You can also contact the trial center directly.
Aurora is currently open at the following locations:
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Sue Anschutz-Rogers Eye Center, University of Colorado - Dept. of Ophthalmology
Aurora, Colorado, United States, 80045
Contact: Rachel Gerle | 720-848-5096
RACHEL.GERLE@CUANSCHUTZ.EDU
Principal Investigator: M. MathiasVitreoRetinal Associates
Gainesville, Florida, United States, 32607
Contact: Jing Zhang | 352-300-8412
Jing@vra-pa.com
Principal Investigator: C KayShriners UK Ophthalmology - University of Kentucky
Lexington, Kentucky, United States, 40536
Contact: Michele Reg | 859-323-5868
Mreg4@uky.edu
Principal Investigator: R MaldonadoCasey Eye Institute, OHSU
Portland, Oregon, United States, 97239
Contact: Kimberly Voelker | 503-494-0020
ordc@ohsu.edu
Principal Investigator: M PennesiRetina Foundation of the Southwest
Dallas, Texas, United States, 75231
Contact: Tein Luu | 214-363-3911 ext 162
tluu@retinafoundation.org
Principal Investigator: D Birch
The above information is also available on worldwide clinical trial database clinicaltrials.gov with identifier (NCT number): NCT04123626.
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.
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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.
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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.
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Yes, travel and accommodation costs will be covered.
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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.
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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.
Ask a question
If you have any questions, please contact us using the form below. You can also mail directly to patientinfo@proqr.com.