An open-label, Multiple Ascending Dose Study to Evaluate the Safety andTolerability of Sepofarsen in Pediatric Subjects of Age 3 to <8 Years withLeber Congenital Amaurosis Type 10 (LCA10) due to the c.2991+1655A>G (p.Cys998X) mutation

ProQR Therapeutics0 个研究点目标入组 10 人开始时间: 2020年9月23日最近更新: 2021年1月5日

适应症

试验速览

暂无简介。

•1. Male or female child, 3 to <8 years of age at Screening
•2. A clinical diagnosis of LCA and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A>G mutation in the CEP290 gene, based on genotyping analysis at Screening. Historic genotyping results from a certified laboratory are acceptable with Sponsor approval.
•3. Best corrected visual acuity (BCVA) equal to or better than Light Perception, and equal to or worse than approximate Snellen equivalent 20/50 in the treatment eye.
•4. Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as determined by the Investigator.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•1. Presence of additional homozygous or compound heterozygous pathogenic mutations (other than the c.2991+1655A>G mutation in the CEP290 gene) in genes associated with other recessive inherited retinal degenerative diseases or syndromes (eg, Usher syndrome) based on genotyping analysis.
•2. Presence of any significant ocular or non-ocular disease/disorder (including medication abnormalities) which may either put the subject at risk because of participation in the trial, may influence the results of the trial, or the subject’s ability to participate in the trial.
•3. Receipt within 3 months prior to Screening of any intraocular or periocular surgery (including refractive surgery), or an intravitreal (IVT) injection or planned intraocular surgery or procedure during the course of the trial.
•4. Current treatment or treatment within the past 3 months or planned treatment with drugs known to be toxic to the lens, retina, or the optic nerve.
•5. Use of any investigational drug or device within 3 months or 5 half-lives of Day 1, whichever is longer, or plans to participate in another study of a drug or device during the trial period.
•6. Any prior receipt of genetic or stem-cell therapy for ocular or non-ocular disease.

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