Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0 Level: PT Classification code 10038914 Term: Retinitis pigmentosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
ProQR Therapeutics IV B.V.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - An adult (= 18 years) willing and able to provide informed consent for participation prior to performing any study related procedure. OR A minor 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. 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 74 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: - 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of QR-421a;Secondary Objective: To evaluate the safety and tolerability of QR-421a To evaluate changes in Patient-Reported Outcome (PRO) measures in subjects treated with QR-421a To evaluate systemic exposure of QR-421a;Primary end point(s): Change from baseline in best corrected visual acuity (BCVA) (based on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart) at 18 months of treatment versus sham-procedure;Timepoint(s) of evaluation of this end point: At multiple timepoints up to 18 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change from baseline in the following outcome measures: Other measures of BCVA Spectral domain optical coherence tomography (SD-OCT) Low Luminance Visual Acuity (LLVA) Microperimetry Static perimetry Full-field Stimulus Threshold (FST) Change from baseline in PRO measures, as assessed by: Veteran Administration Low Vision Visual Functioning Questionnaire (VA LV VFQ-20) (Stelmack 2007) Patient Global Impressions of Severity (PGI-S) Patient Global Impressions of Change (PGI-C) Ocular and non-ocular adverse events (AEs) Exposure of QR-421a in serum;Timepoint(s) of evaluation of this end point: At multiple timepoints up to 24 months | — |
Countries
Brazil, Canada, European Union, Germany, Norway, United Kingdom, United States
Contacts
Public ContactClinical Trial Manager
ProQR Therapeutics IV B.V.
Outcome results
None listed