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Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy (NYRVANA)

2026年9月2日 更新者：SparingVision

A Phase I/IIa Clinical Trial to Assess the Safety, Tolerability, and Efficacy of a Single Intravitreal Injection of SPVN20 Gene Therapy in Participants With Advanced Rod-Cone Dystrophy

This open-label, multi-center study is to evaluate the safety, tolerability, and efficacy of escalating doses of a gene therapy called SPVN20 administered via a single intravitreal injection in participants with advanced Rod-Cone Dystrophy.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

This is an open-label, non-randomized, multi-center, dose-escalation and dose-expansion, first-in-human, Phase I/IIa study. Participants will be followed for a total of five years after receiving a single unilateral intravitreal injection of SPVN20 in their study eye, and will be monitored by an independent Data Safety Monitoring Committee. Eligible patients will be assigned by sequential enrollment to one of the following three cohorts:

Cohort 1: low dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 2: medium dose of SPVN20 will be evaluated in a total of 9 participants. Cohort 3: high dose of SPVN20 will be evaluated in a total of 9 participants.

研究类型

介入性

注册 (估计的)

27

阶段

  • 阶段2
  • 阶段1

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

  • 比利时
      • Ghent、比利时
        • 招聘中
        • Ghent University Hospital
        • 接触：
          • Ophthalmic Clinical Trials Unit
          • 电话号码：00 32 9 332 23
          • 邮箱：OCTU@uzgent.be
        • 首席研究员：
          • Prof. Dr. Bart Leroy
  • 法国
      • Paris、法国、75012
        • 招聘中
        • CHNO XV-XX Paris - CIC 1423
        • 接触：
        • 首席研究员：
          • Dr Amine Benadji
  • 爱尔兰
      • Dublin、爱尔兰
        • 招聘中
        • Royal Victoria Eye and Ear Hospital
        • 接触：
        • 首席研究员：
          • Dr Emma Duignan

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

描述

Inclusion Criteria:

  1. Age ≥ 18 years old at enrollment.
  2. Participant with a genetically confirmed clinical diagnosis of advanced RCD in both eyes due to non-syndromic RCD.
  3. Participants enrolled in the Dose-Escalation cohorts must have a BCVA meeting the study eye criteria.
  4. Documented preservation of foveal cone cell bodies in the study eye (as shown on SD-OCT imaging).
  5. Participant with a history of formed vision.
  6. Participant willing and able to provide informed consent.

Exclusion Criteria:

  1. Participant participating in another clinical trial and receiving an investigational medicinal product (IMP) within either 5 half-lives of that IMP, or 90 days prior to the injection of SPVN20.
  2. Participant with cortical visual impairment.
  3. Participant with systemic disease or other pathology not related to their diagnosis of RCD, and whose symptoms or associated treatments may affect vision.
  4. Participant with known allergies to corticosteroids, or who will be unable to tolerate the corticosteroid regimen.
  5. Participant who has received immunosuppressive therapies or any other therapy known to impact the immune system during the last month prior to SPVN20 administration.
  6. Active ocular inflammation or recurrent history of idiopathic or autoimmune-associated uveitis.
  7. Participant known to be allergic to any of the delivery vehicle constituents or to any other drugs planned to be used during the clinical study.
  8. Active alcohol or substance abuse.
  9. Participant positive for human immunodeficiency virus (HIV) or any other systemic immunocompromising disease.
  10. Participant with active Hepatitis B or Hepatitis C.
  11. Female participant currently pregnant or breastfeeding or intending to become pregnant.
  12. Participant with clinically active ocular infection of herpetic diseases.
  13. Participant with active coronavirus disease (COVID-19) infection.
  14. Participant who received any vaccination/immunization within 28 days prior to screening and/or during screening.
  15. Participant who previously received any gene therapy product, stem cell therapy, cell-based therapy for ocular or non-ocular disease.
  16. Participant with a retinal implant at enrollment.
  17. Participant with pre-existing eye conditions that would interfere with the interpretation of study endpoints, or increase the risk of surgical complications.
  18. Participant with significant media opacity impacting evaluation of the retina or vitreous.
  19. Participant who had intraocular surgery within 90 days of study treatment administration.
  20. Participant with history of corticosteroid-induced raised IOP of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.
  21. Participant with history of unresolved rhegmatogenous retinal detachment in the fovea.
  22. Participant with history of unresolved ocular hypotony.
  23. Participant unwilling or unable (based on the Investigator's judgment) to comply with the study protocol.
  24. Participant with any condition that would not allow them to complete follow-up examinations during the study and, in the opinion of the Investigator, would make them unsuitable for the study.
  25. Participant currently taking restricted classes of medications listed in protocol
  26. Participant with ongoing cardiac or neurological disorders not managed by a restricted medication can be excluded based on the Investigator's judgement.
  27. Women of childbearing potential (WOCBP) and men and/or their partner(s) of childbearing potential who do not agree to use a highly effective contraceptive method.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：非随机化
  • 介入模型：顺序分配
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：SPVN20 (low dose). Single intravitreal injection of a low dose of SPVN20 on Day 0
SPVN20
生物：SPVN20 (low dose)
SPVN20
实验性的：SPVN20 (medium dose). Single intravitreal injection of a medium dose of SPVN20 on Day 0
SPVN20
生物：SPVN20 (medium dose)
SPVN20
实验性的：SPVN20 (high dose). Single intravitreal injection of a high dose of SPVN20 on Day 0
SPVN20
生物：SPVN20 (high dose)
SPVN20

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Safety and Tolerability
大体时间：6 months
Incidence and severity of ocular and non-ocular treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
6 months
Ocular safety
大体时间：6 months
General ocular assessments including complete ophthalmic examination of extraocular and intraocular structures
6 months

次要结果测量

结果测量
措施说明
大体时间
Best Corrected Visual Acuity (BCVA)
大体时间：6 months
Change in BCVA from Baseline to Month 6 after dosing with SPVN20.
6 months
Full-field stimulus threshold (FST) test
大体时间：6 months
Change in FST from Baseline to Month 6 after dosing with SPVN20
6 months

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

SparingVision

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2025年10月8日

初级完成 (估计的)

2032年3月1日

研究完成 (估计的)

2032年3月1日

研究注册日期

首次提交

2026年8月10日

首先提交符合 QC 标准的

2026年9月2日

首次发布 (实际的)

2026年9月8日

研究记录更新

最后更新发布 (实际的)

2026年9月8日

上次提交的符合 QC 标准的更新

2026年9月2日

最后验证

2026年9月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • SPVN20-CLIN-01
  • 2025 (美国 NIH 拨款/合同：Faculty of Social Sciences Scientific Grant at the University of Gdańsk)
  • 2025-520665-47-00 (克蒂斯)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

未定

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

在美国制造并从美国出口的产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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