A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety, Tolerability and Systemic Exposure of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene - Illuminate

ProQR Therapeutics0 个研究点目标入组 30 人开始时间: 2019年1月24日最近更新: 2019年12月10日

适应症

试验速览

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•1.Male or female, = 8 years of age at Screening with a clinical diagnosis of LCA and a molecular diagnosis of homozygosity or compound heterozygosity for the CEP290 p.Cys998X mutation, based on genotyping analysis at Screening. Historic genotyping results from a certified laboratory are acceptable with Sponsor approval.
•2.Detectable outer nuclear layer (ONL) in the area of the macula as determined by the reading center at Screening.
•3.An ERG result consistent with LCA, as determined by the reading center. A historic ERG result may be acceptable for eligibility
•4.Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 13
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 17
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1.Any contraindication to IVT injection according to the Investigator’s clinical judgment and international guidelines (Avery 2014)
•2.Any ocular and/or general disease or condition that could compromise subject’s safety or interfere with assessment of efficacy and safety, as determined by the Investigator
•3.Prior receipt of intraocular surgery or IVT injection within 3 months prior to study start or planned intraocular surgery or procedure during the course of the study
•4.Use of any investigational drug or device within 90 days or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the PQ 110-003 study period
•5.Any prior receipt of genetic therapy for LCA

发起方