QR-421a Clinical trial: STELLAR

Phase 1/2 "Safety and efficacy study" (ongoing)
STELLAR is an ongoing international Phase 1/2, single dose, double-masked, randomized, sham-controlled clinical trial of QR-421a in patients with Usher syndrome type 2 or non-syndromic retinitis pigmentosa due to mutation(s) in exon 13 of the USH2A gene. QR-421a is an investigational drug that is designed to stop the progression and potentially reverse the vision loss associated with these diseases.

Approximately 18 adults will be included in STELLAR. During the study, patients will receive a single intravitreal injection of one of the three dose levels of QR-421a in one eye or a sham-procedure. STELLAR will be conducted at about seven expert clinical centers in North America and select European countries. Unfortunately not everyone who is eligible and interested may be able to participate. Please check this website if you would like to stay informed on our progress and potential future trial participation opportunities.

The purpose of the STELLAR study is to find out if different dose levels (amounts) of QR-421a given as a single intravitreal injection in one eye, are safe in patients with Usher syndrome type 2 or RP due to mutations in exon 13 of the USH2A gene. This study will also measure whether QR-421a results in any improvements to eyesight (visual field and visual acuity) or retinal structure (optical coherence tomography). The study will also help us to gather information on the frequency of treatment. Interim results from this first-in-human study are expected in the first quarter of 2020. Participants completing this trial will be able to participate in an extension study if eligible.



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

Additional details about STELLAR and where it is being conducted are available at www.clinicaltrials.gov and will be explained in detail by physicians conducting the trial.
What is a Phase 1/2 trial?
A trial may include more than one phase. A phase 1/2 clinical trial tests the safety of an investigational drug, as well as assesses signals of efficacy.

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