An Open-Label, Single Arm, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects with Leber’s Congenital Amaurosis (LCA) due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

ProQR Therapeutics0 个研究点目标入组 12 人开始时间: 2017年4月13日最近更新: 2019年12月16日

适应症

试验速览

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•1. Male or female, = 6 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.
•2. Best-corrected visual acuity greater than or equal to light perception in both eyes and equal to or worse than LogMAR + 1.0 (Snellen notation 20/200) in the worse eye and equal to or worse than LogMAR + 0.7 (Snellen notation 20/100) in the contralateral eye.
•3. Detectable outer nuclear layer (ONL) in the area of the macula.
•4. An ERG result consistent with LCA.
•5. Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 6
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 5
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

•1. Syndromic disease
•2. Pregnant or breast-feeding female
•3. Any clinically significant cardiac disease or defect.
•4. One or more coagulation parameters outside of the normal range.
•5. Any ocular disease or condition that could compromise treatment safety, visual acuity or interfere with assessment of efficacy and safety.
•6.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
•7. 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-110-001 study period.
•8. Any prior receipt of genetic therapy for LCA

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