ProQR RNA Therapy Trial for Patients with Autosomal Dominant Retinitis Pigmentosa

A Prospective First-In-Human Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects with Autosomal Dominant Retinitis Pigmentosa (adRP) due to the P23H Mutation in the RHO Gene

The purpose of this study is to learn more about an investigational drug, QR-1123 (referred to as “the study drug”), and to find out if the study drug is safe at different dose levels. The study will also measure whether the study drug improves your vision. The study drug is given by something called an “intravitreal injection”, which is an injection in your eye.

There will be 2 groups for this study: (1) a single dose cohort, and (2) a repeat dose cohort. Everyone in the single dose cohort will receive the study drug through an injection in their worse-seeing eye. If you are in a repeat dose cohort, you will be randomly assigned, like the roll of a dice, to either the study drug or the sham procedure. Some participants will receive no injection, because the study drug will be compared to no injection. This is called a sham procedure. The sham procedure will be almost the same as the injection procedure, but you will not get an injection in your eye. You will have a 3/4 chance of receiving the study drug and a 1/4 chance of receiving the sham procedure. You and the study team will not know if you received the study drug or the sham procedure, but we are able to find out in the case of an emergency.

Some of the requirements for this study are:

  • At least 18 years or older
  • Have a clinical presentation consistent with autosomal dominant Retinitis Pigmentosa
  • Must have the P23H mutation in the RHO gene. All subjects will have genetic testing completed for the study
  • Impaired visual field with some central vision
  • Good general health

This study will occur at the Casey Eye Institute in Portland, OR. Participation in this study will last approximately 14 months (including the screening period). You will have ten (10) visits if you’re in a single dose cohort, and thirteen (13) visits if you’re in a repeat dose cohort. Participants in the repeat dose cohort will also have three (3) phone visits, where we review your health and medications. Study visits will involve visual acuity testing, dilated and undilated visual field tests, and blood draws.

For more information, please call the study coordinator at 503-494-0020, or email at

Principal Investigator:
Mark Pennesi, MD, PhD
Casey Eye Institute
Oregon Health & Science University
3375 SW Terwilliger Blvd
Portland, OR 97239
eIRB#: 20144