NL-OMON55235已完成2 期
An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age with Leber Congenital Amaurosis Type 10 (LCA10) due to the c.2991 +1655A>G (p.Cys998X) mutation - PQ-110-005
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 3
研究概览
简要总结
Trial is onging in other countries
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 11（—）
入选标准
- •1. Male or female child, <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. 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.
排除标准
- •1. Presence of pathogenic mutations in genes associated with other recessive,
- •dominant or X-linked inherited retinal degenerative diseases or syndromes based
- •on genetic 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 1 month 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 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.
研究者
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