QR-421a Clinical trial: Stellar

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

Unfortunately not everyone who is eligible and interested may be able to participate. Please sign up for the Eye Connect newsletter or follow us on social media if you would like to stay informed on our progress and potential future trial participation opportunities.

Positive interim study findings and next steps
Three-months interim findings from Stellar, reported in March 2020, suggested QR-421a given as a single intravitreal injection was safe and well tolerated. Two of eight participants in the treated groups responded to QR-421a treatment thus far showing benefit in multiple outcome measures including retinal structure and visual function. A similar response was not observed in the six participants included in the control (“sham procedure”) group.

Based on these early positive findings we will continue the trial as designed with two additional study groups testing different dose levels (amounts) of QR-421a.

Trial design and purpose of the study
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. Participants receive a single intravitreal injection of one of the three dose levels of QR-421a in one eye or a sham-procedure. Stellar is conducted at about seven expert clinical centers in North America and select European countries. This study will also measure whether QR-421a results in any improvements to vision (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. Participants completing this trial will be able to participate in an extension study if eligible.

Stellar Study design infographic. Phase 1/2 single dose trial and 24 months follow up. Three groups (low, mid and high dose) are shown in a staggered way (teal bars), each starting some time after the other. Arrows from the three groups point to an extension treatment study (purple bar) *depending on risk/benefit analysis. In this study sham procedure group also to receive study drug.

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.