Clinical Trials for Individuals with USH2A-related Retinitis Pigmentosa

ProQR Therapeutics has partnered with InformedDNA to assist in locating individuals who may be eligible for the Sirius clinical trial aimed at treating retinitis pigmentosa and Usher syndrome caused by specific mutations in the USH2A gene. InformedDNA’s genetic counselors are highly knowledgeable about inherited retinal diseases, and can answer questions and review eligibility to enable individuals to make more informed decisions about clinical trial participation.

Speaking with a genetic counselor at InformedDNA about these studies is provided at no cost to the individual. After speaking with a genetic counselor, individuals interested in learning more about a clinical research study will be connected with a trial site closest to them.

Information about the clinical trial sponored by ProQR Therapeutics

Sirius is the name of the clinical trial that aims to find out whether the investigational RNA therapy ultevursen (previously named QR-421a) is effective and safe for people with retinitis pigmentosa and Usher syndrome due to mutations in exon 13 of the USH2A gene. This clinical trial has been approved by the FDA, following results from a Phase 1/2 clinical trial of this same therapy.

What is Ultevursen (QR-421a)?

Ultevursen (QR-421a) is an investigational therapy that is designed to skip over the exon 13 region in RNA which carries mutations that lead to retinitis pigmentosa and Usher syndrome. The aim of ultevursen is to stop vision loss in people with this condition.

In the first clinical trial of ultevursen (QR-421a), the Stellar study, a benefit was observed in the ultevursen (QR-421a) treated eyes in comparison to the untreated eyes across multiple vision tests. Ultevursen (QR-421a) was also observed to be well tolerated with no serious adverse events reported during the trial.

How is the clinical trial set up?

The Sirius study is designed for people, 12 years of age or older, with advanced vision loss due to retinitis pigmentosa and Usher syndrome with mutations in exon 13 of the USH2A gene. The study aims to find out whether ultevursen (QR-421a) is effective at stopping vision loss and whether it is safe and well-tolerated. 

To request an appointment with a Genetic Counselor at InformedDNA to further discuss this trial opportunity

  • Use our convenient online form to request an appointment
  • Or, call 888-910-1015 to speak with a member of our Patient Care team

What to expect in the appointment with a Genetic Counselor

The genetic counselors at InformedDNA working on this program are highly trained in ophthalmology genetics. Our appointments occur over the phone, so family members or friends may participate in the appointment, regardless of location. Appointments are available during usual business hours, as well as evenings and weekends.

Some of the questions that may be addressed during the call include:

  • Inclusion/exclusion criteria (i.e., what makes you a possible candidate for this clinical trial?)
    • The genetic counselor will review basic inclusion and exclusion criteria to determine if you meet the baseline eligibility for the trial.
    • If you have current visual field (VF) and best corrected visual acuity (BCVA) test results, please have them during your appointment with the genetic counselor. If you do not have current VF and BCVA test results, you may need further testing at one of the trial sites to determine eligibility. Testing at a trial site to confirm eligibility for the study is done at no cost to you.
    • The trial site will have additional inclusion/exclusion criteria to review with potential participants.
  • Definitions and expectations of different phases of clinical trials (phase 1, phase 2, etc)
  • Differences between therapies (ex: gene therapies, RNA therapy, stem cell therapy, CRISPR)
  • Review of genetic cause of disease and gene-based approaches to therapy
    • The genetic counselor will review prior genetic test results to ensure you meet the genetic eligibility criteria for the study. It is helpful if you provide these results in advance of your appointment with the genetic counselor, either by uploading them to the appointment request form, faxing (760-203-1194), or by uploading them to the InformedDNA patient portal after scheduling.

Additional Information

Please click this link for additional information from ProQR on the Sirius clinical trial.

This service with InformedDNA is sponsored by ProQR Therapeutics. No identifying information of individuals will be shared with ProQR Therapeutics.

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