EUCTR2021-002728-19-NO进行中（未招募）1 期
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 Early to Moderate Vision Loss - Celeste
ProQR Therapeutics IV B.V.0 个研究点目标入组 120 人开始时间: 2021年10月19日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 120
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •An adult (= 18 years) willing and able to provide informed consent for participation prior to performing any study related procedure. OR
- •A minor with a parent or legal guardian willing and able to provide written permission for the subject’s participation prior to performing any study related procedures and pediatric subjects able to provide age appropriate assent for study participation.
- •- 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 113
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •- 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.
研究者
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