EUCTR2018-003501-25-IT进行中（未招募）1 期
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
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
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
- •Relating to Study Initiation : The subject is eligible for the study and thus eligible to receive QR-110 or sham-procedure in the treatment eye (ie, the first eye to be treated) if all the following inclusion criteria apply at Screening/Day 1:
- •1. An adult ( = 18 years) willing and able to provide informed consent for participation -OR- a minor (8 to < 18 years) 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.
- •2. 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.
- •3. BCVA better or equal to Logarithm of the Minimum Angle of Resolution (logMAR) +3.0 (Hand Motion), and equal or worse than logMAR + 0.4 (approximate Snellen equivalent 20/50) in the treatment eye, using the best BCVA reading at Screening and based on the Early Treatment Diabetic Retinopathy Study (ETDRS) or the Berkeley Rudimentary Vision Test (BRVT).
- •4. Detectable outer nuclear layer (ONL) in the area of the macula as determined by the Investigator at Screening.
- •5. An electroretinogram (ERG) result consistent with LCA, as determined by the Investigator. A historic ERG result may be acceptable for eligibility.
- •6. Clear ocular media and adequate pupillary dilation to permit good quality retinal imaging, as assessed by the Investigator.
- •7. Non-pregnant and non-breastfeeding subjects.
- •Relating to Treatment Initiation Contralateral Eye:
- •1. BCVA better or equal to Logarithm of the Minimum Angle of Resolution (logMAR) +3.0 (Hand Motion), and equal or worse than logMAR + 0.4 (approximate Snellen equivalent 20/50) in the contralateral eye, using the best BCVA reading at Month 12 (see Section 8.1) and based on the Early Treatment Diabetic Retinopathy Study (ETDRS) or the Berkeley Rudimentary Vision Test (BRVT).
- •2. Detectable outer nuclear layer (ONL) in the area of the macula of the contralateral eye as determined by the Investigator.
- •3. Clear ocular media and adequate pupillary dilation to permit goodquality retinal imaging in the contralateral eye, as assessed by the Investigator.
- •4. Non-pregnant and non-breastfeeding subjects.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •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
排除标准
- •Relating to Study Initiation : The subject is ineligible for the study if any of the following criteria apply at Screening/Day 1:
- •1. Presence of any significant ocular or non-ocular disease/disorder (including medication and 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.
- •2. 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 study period.
- •3. Any prior receipt of genetic or stem-cell therapy for ocular or non ocular disease.
- •4. Receipt within 3 months prior to Screening of any intraocular or periocular surgery (including refractive surgery), or an IVT injection or planned intraocular surgery or procedure during the course of the study.
- •5. Known hypersensitivity to antisense oligonucleotides or any constituents of the injection.
- •6. Pregnant and breastfeeding subjects.
- •Relating to Treatment Initiation Contralateral Eye:
- •1. Presence of any significant ocular or non-ocular disease/disorder (including medication and 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. This includes but is not limited a subject who: 1) is not an appropriate candidate for antisense oligonucleotide treatment, 2) has concurrent cystoid macular edema (CME) in the contralateral eye.
- •2. A planned IVT injection or intraocular or periocular surgery/procedure (including refractive surgery) in the contralateral eye during the course of the study.
- •3. Plans to participate in another study of a drug or device during the study period.
- •4. Pregnant and breastfeeding subjects.
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