NCT05417126已完成2 期
A Phase 2a, Open Label Multicenter Clinical Trial to Evaluate the Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt Disease
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 3
- 主要终点
- Type, severity, and incidence of ocular and systemic adverse events (AEs)
研究概览
简要总结
The purpose of the study is to evaluate the safety and effects of a single intravitreal injection of virally-carried Multi-Characteristic Opsin (vMCO-010) in Subjects with Stargardt Disease
详细描述
This multicenter open label study will evaluate single dose level of vMCO-010 in up to 6 subjects with Stargardt's Disease. Subjects with documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM 1. All subjects will continue to be assessed for 48 weeks following treatment with vMCO-010.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥16 years of age
- •Able to comprehend and give informed consent.
- •Able to comply with testing and all protocol tests.
- •Documented clinical diagnosis of Stargardt disease (classic fleck phenotype and/or well-demarcated sub-foveal area of significantly reduced autofluorescence as imaged by FAF), or genetic diagnosis with pathogenic variants in ABCA4, ELOVL4, or PROM1
- •In the study eye: ETDRS BCVA in range of 1.3 logMAR (Approximate Snellen equivalent: 20/400) to 1.9 logMAR (Snellen equivalent: 20/1600), and ETDRS BCVA no better than 20/200 in the fellow eye.
- •Presence of retinal inner nuclear and nerve fiber layers on optical coherence tomography (OCT) testing in the study eye at screening
排除标准
- •Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).
- •Received any of the following treatments: gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips (such as ARGUS-II) or sub-retinal injections.
- •Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
- •Participation in an interventional study of a vitamin A derivative ≤ 3 months prior to screening
- •Presence of significant cardiovascular or cerebrovascular disease, including stroke within 12 months of entry.
- •Resting heart rate outside specified limits upon repeated measurement.
- •History of uncontrolled diabetes, hepatitis, pancreatitis, cirrhosis, liver failure, uncontrolled thyroid disease or hypervitaminosis A.
- •Any intraocular surgery or thermal laser within 3 months of trial entry or any prior thermal laser in the macular region.
- •Any major surgical procedure within one month of trial entry or anticipated during the trial.
- •Clinically significant abnormal lab results at screening
- •Known serious allergies to the fluorescein dye used in angiography or intraocular pressure measurement, povidone iodine, or to the components of the vMCO-010 formulation
- •In the Investigator's opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding or laboratory abnormality
- •Pre-existing conditions in the study eye such as glaucoma, diseases affecting the optic nerve causing significant visual field loss, history of uveitis, corneal or lenticular opacities).
- •Presence of any complicating systemic diseases such as malignancies whose treatment could affect central nervous system function..
- •Subjects who are positive for syphilis, hepatitis B, C, and human immunodeficiency virus (HIV) will be excluded..
- •Presence of narrow iridocorneal angles contraindicating pupillary dilation in the study eye.
- •Presence of disorders of the ocular media in the study eye which could interfere with visual acuity and other ocular assessments, including OCT, during the study period.
- •Presence of macular hole in the study eye, evident by ophthalmoscopy and/or by OCT examinations
- •Current evidence of retinal detachment in the study eye assessed by the Investigator that significantly affects central vision.
- •Current use of hydroxychloroquine, chloroquine, or any related retina-toxic compounds.
- •Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
结局指标
主要结局
Type, severity, and incidence of ocular and systemic adverse events (AEs)
时间窗: 48 weeks
Type, severity, and incidence of ocular and systemic adverse events (AEs), specifically those related to intravitreal injection of vMCO-010
次要结局
- Effect of vMCO-010 on determination of shape(48 Weeks)
- Effect of vMCO-010 on determination of optical flow(48 Weeks)
- Effect of vMCO-010 as assessed by visual acuity(48 weeks)
- Effect of vMCO-010 on Light-guided Mobility(48 Weeks)
研究者
研究点 (3)
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相关资讯
FDA Accepts Nanoscope's BLA for MOGENRY, an Optogenetic Gene Therapy for Severe Vision Loss in Retinitis Pigmentosa- The FDA has accepted and filed Nanoscope Therapeutics' Biologics License Application for MOGENRY (sonpiretigene isteparvovec, MCO-010) in retinitis pigmentosa with severe vision loss.
- The filing is supported by the Phase 1/2a trial and the RESTORE Phase 2b/3 sham-controlled trial, which met its primary and key secondary endpoints at weeks 52 and 76.
- MOGENRY is a one-time, in-office intravitreal optogenetic gene therapy that requires no genetic testing or surgical suite and showed no treatment-related serious adverse events.
- If approved, MOGENRY would become the first gene-agnostic therapy to improve vision in RP patients with severe vision loss, who currently have no approved therapy.7 days agoNanoscope's MCO-010 Receives FDA RMAT and Five EMA Orphan Designations for Retinal Dystrophies- The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to MCO-010 for Stargardt disease, building on prior Orphan Drug and Fast Track designations for both retinitis pigmentosa and Stargardt disease.
- The European Medicines Agency awarded five Orphan designations to MCO-010 covering non-syndromic and syndromic rod-dominant and cone-dominant dystrophies, as well as macular dystrophies.
- MCO-010 is a one-time intravitreal optogenetic therapy that activates bipolar retinal cells to restore vision without requiring genetic testing or surgical intervention.
- Nanoscope has initiated a rolling BLA submission following positive Phase 2b/3 RESTORE trial results in retinitis pigmentosa and plans Phase 3 trials for Stargardt disease in 2025.last yearNanoscope Therapeutics Showcases MCO-010 Optogenetic Therapy Data at ASGCT 2025- Nanoscope Therapeutics presented multiple research presentations at ASGCT 2025 highlighting the safety and efficacy of MCO-010 optogenetic therapy for retinal degenerative diseases.
- The company reported positive therapeutic benefits in Stargardt disease patients similar to durable vision gains previously observed in retinitis pigmentosa patients.
- MCO-010 represents the first mutation-agnostic therapy shown to restore vision in advanced RP patients via a one-time intravitreal injection.
- The company plans to initiate BLA submission for MCO-010 to treat RP in 2025 following positive Phase 2b RESTORE trial results.last yearNanoscope Plans BLA Submission for MCO-010 Gene Therapy in Retinitis Pigmentosa- Nanoscope Therapeutics plans to submit a Biologics License Application (BLA) to the FDA in Q1 2025 for MCO-010 to treat retinitis pigmentosa.
- MCO-010, a gene-agnostic optogenetic therapy, has shown significant vision improvement in advanced RP patients in Phase 2b/3 RESTORE trial.
- New data shows MCO-010 arrests retinal degeneration in animal models, suggesting a disease-modifying effect in addition to vision restoration.
- The FDA has granted Fast Track designation to MCO-010 for both RP and Stargardt disease, and a Phase 3 trial for Stargardt is planned.last yearNanoscope Therapeutics to Advance MCO-010 for Stargardt Disease to Phase 3 Trial Following FDA Meeting- Nanoscope Therapeutics' MCO-010 gene therapy is set to advance to a Phase 3 registrational trial for Stargardt Macular Degeneration after a productive meeting with the FDA.
- The FDA has provided input on the Phase 3 trial design, which will assess MCO-010's safety and efficacy using best-corrected visual acuity as the primary endpoint.
- The planned Phase 3 trial will enroll legally blind patients as young as 12 years old, expanding the population from the Phase 2 study, and will be the first randomized, controlled gene therapy trial for Stargardt disease.
- The FDA has agreed that the current nonclinical data package is sufficient to support a future Biologics License Application (BLA) submission for MCO-010.2 years ago