A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss
- Conditions
- progressive pigmentary retinopathyrod-cone dystrophyRP1001592010010463
- Registration Number
- NL-OMON52147
- Lead Sponsor
- ProQR Therapeutics IV B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 15
- An adult (>= 18 years) willing and able to provide informed consent for
participation prior to performing any study related procedure. OR A minor
(>12<18) able to complete all study assessments and comply with the protocol
and has a parent or caregiver willing and able to follow study instructions,
and attend study visits with the subject as required.
- 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 for 1 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.
- 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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>- Change from baseline in best corrected visual acuity (BCVA) (based on the<br /><br>Early Treatment Diabetic Retinopathy Study (ETDRS) chart) at 18 months of<br /><br>treatment versus sham-procedur</p><br>
- Secondary Outcome Measures
Name Time Method <p>- Change from baseline in the following outcome<br /><br>measures:<br /><br>Other measures of BCVA<br /><br>Spectral domain optical coherence tomography<br /><br>(SD-OCT)<br /><br>Low Luminance Visual Acuity (LLVA)<br /><br>Microperimetry<br /><br>Static perimetry<br /><br>Full-field Stimulus Threshold (FST)<br /><br><br /><br>- Change from baseline in PRO measures, as assessed<br /><br>by:<br /><br>Veteran Administration Low Vision Visual<br /><br>Functioning Questionnaire (VA LV VFQ-20)<br /><br>Patient Global Impressions of Severity (PGI-S)<br /><br>Patient Global Impressions of Change (PGI-C)<br /><br>- Ocular and non-ocular adverse events (AEs)<br /><br>- Exposure of QR-421a in serum</p><br>