A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss
- Conditions
- Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A GeneMedDRA version: 20.0Level: PTClassification code 10038914Term: Retinitis pigmentosaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Eye Diseases [C11]
- Registration Number
- EUCTR2021-002728-19-FR
- Lead Sponsor
- ProQR Therapeutics IV B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 120
- An adult (= 18 years) willing and able to provide informed consent for participation prior to performing any study related procedure. OR A minor with a parent or legal guardian willing and able to provide written permission for the subject’s participation prior to performing any study related procedures and pediatric subjects able to provide age appropriate assent for study participation.
- Clinical presentation consistent with RP with Usher syndrome type 2 or non-syndromic form of RP (NSRP), based on ophthalmic, audiologic, and vestibular examinations.
- A molecular diagnosis of homozygosity or compound heterozygosity for1 or more pathogenic exon 13 mutations in the USH2A gene, based on genetic analysis at screening.
- Reliable BCVA, perimetry, and other measurements in both eyes.
Are the trial subjects under 18? yes
Number of subjects for this age range: 5
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 113
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2
- Presence of any significant ocular or non-ocular disease/disorder (or medication and/or laboratory test abnormalities) which, in the opinion of the Investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or the subject's ability to participate in the study.
- Known hypersensitivity to antisense oligonucleotides or any constituents of the injection.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of QR-421a;Secondary Objective: To evaluate the safety and tolerability of QR-421a<br>To evaluate changes in Patient-Reported Outcome (PRO) measures in subjects treated with QR-421a<br>To evaluate systemic exposure of QR-421a;Primary end point(s): Change from baseline in mean sensitivity (based on <br>static perimetry) at 12 months of treatment versus sham-procedure<br>;Timepoint(s) of evaluation of this end point: At multiple timepoints up to 12 months
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Change from baseline in the following outcome <br>measures:<br> BCVA <br> Spectral domain optical coherence tomography <br>(SD-OCT)<br> Low Luminance Visual Acuity (LLVA)<br> Other measures of static perimetry <br> Full-field Stimulus Threshold (FST)<br>Change from baseline in PRO measures, as assessed <br>by:<br> Michigan Retinal Degeneration Questionnaire <br>(MRDQ) (Lacy 2021)<br> Patient Global Impressions of Severity (PGI-S)<br> Patient Global Impressions of Change (PGI-C)<br>Ocular and non-ocular adverse events (AEs)<br>Exposure of QR-421a in serum;Timepoint(s) of evaluation of this end point: At multiple timepoints up to 24 months