QR-110 Clinical trial: Study 001

Phase 1/2 "Safety and efficacy trial"
Clinical Study 001 is an ongoing international Phase 1/2, open-label, safety, tolerability and efficacy trial of QR-110 in Leber’s congenital amaurosis 10 (LCA 10) patients carrying one or two copies (compound heterozygous or homozygous respectively) of the p.Cys998X CEP290 gene mutation (also known as the c.2991+1655A>G mutation). QR-110 is an investigational drug that is designed to be administered by injections into the vitreous of the eye (intravitreal injections).

The patients that participate in study 001 will receive an intravitreal injection of QR-110 into one eye every three months for one year (in total each patient will receive 4 injections). Approximately 6 adults (at least 18 years old) and 6 children (6-17 years old) will participate in this study. The trial is planned to be conducted at three academic hospitals in the USA and Europe.

The primary objective of the study is to test whether QR-110 injections are safe and tolerated. Secondary objectives are to test for the restoration/improvement of visual function (efficacy) and retinal structure measured through specialized ophthalmic tests, and the QR-110 pharmacokinetics. Changes in quality of life will also be evaluated.

For questions, please contact ProQR Therapeutics Patient Advocacy via the contact form or email patientinfo@proqr.com.

Additional details about this clinical trial are available at www.clinicaltrials.gov and will be explained in detail by physicians conducting the study. Below is a list of participating academic hospitals.
What is a Phase 1/2 trial?
A trial may include more than one phase. A phase 1/2 clinical trial tests the safety and identifies the side effects of different doses of an investigational drug, as well as how patients respond to it.

Combining phases 1 and 2 may allow research questions to be answered more quickly or with fewer patients.

Early phase trials are necessary to progress to other types of trials (Phase 3) designed to evaluate the safety and efficacy of an investigational drug over a longer time.

Study Locations

Study 001 is planned to be conducted at the following academic hospitals:

North America

  • University of Iowa, Iowa City, Iowa, United States
  • Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania, United States

  • Ghent University Hospital and Ghent University, Ghent, Belgium, B-9000

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